Biologics Studied and Not
Studied in Children*
To identify biologics that have been studied, were being studied, or were planned for study in children, the Institute of Medicine committee examined several sources of information about biologics that were approved by the Food and Drug Administration (FDA) between January 1, 1997, and December 31, 2010. FDA supplied the list of biologics for products now regulated by the Center for Drug Evaluation and Research (CDER). For the biologics that are regulated by the Center for Biologics Evaluation and Research (CBER), the committee relied on a website listing of biologics for which some supporting documentation was available. CBER staff were consulted to help the committee identify any relevant omitted products and exclude products that were approved under new drug applications (NDAs), were not being marketed, or were not new products.
As explained in Chapter 8, the committee excluded preventive vaccines and nontherapeutic biologics such as assays and reagents (e.g., products used for blood testing or blood grouping). In addition to excluding products approved before 1997, it also excluded products that were approved under new drug applications, were not approved for marketing in the United States, were not being marketed as of December 31, 2010, or were
* Tables were prepared with the assistance of Lara Ellinger, Pharm.D., B.C.P.S., Department of Pharmacy Practice, College of Pharmacy, University of Illinois at Chicago.
not new products.1 The final list included 96 biologics. Of these, 57 were regulated by CDER and 39 were regulated by CBER. This appendix reports information from the labeling of these products and from a government registry of clinical trials.
Although the committee excluded vaccines for its more extensive analysis, it conducted a less intensive review of information on pediatric studies and labeling for vaccines. It identified 55 vaccines with supporting information that CBER had posted at http://www.fda.gov/BiologicsBloodVaccines/ucm133705.htm. The vaccines listed include approximately 20 that appear to have been approved before 1997.
A number of vaccines (e.g., vaccines for rotavirus and combination vaccines for diphtheria, tetanus, and pertussis) are labeled for pediatric use only. Of the 55 vaccines listed by CBER, three products (5 percent) were not labeled for pediatric use, had waivers of pediatric study requirements, and also did not have pediatric studies registered at ClinicalTrials.gov.
• An adenovirus type 4 and type 7 vaccine (no brand name) was developed under contract with the U.S. Department of Defense and approved by FDA in 2011 for use with military personnel ages 17 to 50 years; an earlier product had been used by the military beginning in the 1980s and ending after the sole manufacturer stopped manufacturing the product (Schrager, 2011). FDA waived the pediatric study requirement because studies were impossible or impracticable (Malarkey and Baylor, 2011).2
• An anthrax vaccine (no brand name) was approved in 1970 for use by individuals ages 18 to 65 years who are at high risk of exposure to the disease. In a 2008 approval for a new dosing interval and route of administration, FDA waived the pediatric study requirement on the grounds that studies were impossible or impracticable because the pediatric population is not at high risk of exposure (Sun, 2008).
• A herpes zoster (shingles) vaccine (Zostamax) was approved by FDA in 2006 for use by individuals 60 years of age or older. FDA waived the requirement for pediatric studies because the product
1 The original approval dates and marketing status for biologics are not always easily determined. It is possible that one or more of the products listed had an original approval date prior to 1997. After the release of the prepublication manuscript at the end of February 2012, study staff determined that two products (Peginterferon alfa-2B; ribavirin [Pegintron/Rebetrol combo pack] and methoxy polyethylene glycol-epoetin beta [Mircera]) were not marketed as of December 31, 2010, and that one excluded product (drotrecogin alfa [Xigris]) was not discontinued until 2011. Tables D-1 and D-2 were revised to reflect this information. These revisions did not affect the report’s overall conclusions.
2 References cited in this appendix are included in the report’s reference list.
did not offer a meaningful therapeutic benefit over existing products and was unlikely to be used by a substantial number of children (Baylor, 2006).
In addition, FDA waived pediatric studies (without explanation) in approving a vaccine (Twinrix) for prevention of hepatitis A and B (Richman, 2007). For this product, however, ClinicalTrials.gov lists pediatric studies (see, e.g., ClinicalTrials.gov identifier: NCT00107042). For an intradermal formulation of an influenza vaccine (Fluzone), FDA waived pediatric studies because the product did not offer a meaningful therapeutic benefit over existing products and was unlikely to be used by a substantial number of children (Sun, 2011). ClinicalTrials.gov also lists pediatric studies of this product (see, e.g., ClinicalTrials.gov identifier: NCT00391391).
For the biologics including in the committee’s more extensive investigation, the committee consulted the current product labeling for references to pediatric studies; examined approval letters, if available, for references to required pediatric studies; checked FDA’s tracking database for postmarket study requirements and commitments for required studies; and searched ClinicalTrials.gov. ClinicalTrials.gov is a registry of publicly and privately supported clinical trials that is administered by the National Institutes of Health.
Table D-1, which groups CDER-and CBER-regulated products together, summarizes pediatric information found in the manufacturer’s product labeling. This information includes any pediatric use(s) for which the product is labeled; descriptions in the labeling of pediatric studies of the product (including studies that did not demonstrate efficacy); and, especially for any products without such labeling information, any warnings against pediatric use based on FDA or other analyses of adverse event reports or similar data. Information relevant to use of a product by pediatric populations may be located in several sections of the structured label (e.g., in sections on dosage, clinical pharmacology, and adverse reactions as well as in the highlights section that now appears at the start of prescription labeling). This can complicate efforts to find and summarize this information. Most of the review of labeling occurred in July and August 2011.3
Table D-2 summarizes information about pediatric studies registered at the ClinicalTrials.gov database. It first presents the information for CDER-regulated products and then presents the information for CBER-regulated
3 After the release of the prepublication manuscript, study staff determined that Table D-1 should be revised to categorize two products (basiliximab [Simulect] and digoxin immune Fab [DigiFab]) as labeled for pediatric use. Some other summary information was edited for specificity. These revisions, based on reexamination of the manufacturer’s labeling, did not affect the report’s overall conclusions.
products. For this table, products in certain classes (e.g., intravenous immune globulins) that are often treated as interchangeable for certain uses were grouped together because database entries often did not identify studied products by brand name. Trials for which the lower end of a participant age range was 16 years are not included. The database was checked from July to December 2011.
The brief summaries in the trials database were sometimes incorrect in indicating that a trial included children, particularly when the more detailed trial descriptions did not include an overview description of the age range but did make clear in the inclusion or exclusion criteria that only adults were eligible. These brief summaries could also be misleading about the condition to be studied, for example, by specifying transplantation rather than transplantation-related complications or disorders. A study categorized in the database as a Phase IV study, particularly one requested under the Best Pharmaceuticals for Children Act or required under the Pediatric Research Equity Act, might also fit the definition of a Phase I, II, or III study.
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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1. Abatacept |
• Adult rheumatoid arthritis • Juvenile idiopathic arthritis (JIA) |
Labeled pediatric use(s)
• ≥6 yr with moderately to severely active polyarticular JIA; may be used as monotherapy or concomitantly with methotrexate • Not established in patients <6 yr • Not established for diseases other than JIA Study information • Safety and efficacy were assessed in patients 6 to 17 yr (n = 190). • Findings showed that the risk of disease flare in patients on Orencia was <1/3 the risk for flare in patients withdrawing from Orencia. • Infections were the most frequent adverse events. |
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2. AbobotulinumtoxinA |
• Cervical dystonia • Temporary improvement in glabellar lines |
Cervical dystonia: Safety and effectiveness not established in pediatric patients Glabellar lines: Not recommended for pediatric patients <18 yr |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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3. Adalimumab |
• Rheumatoid arthritis • Juvenile idiopathic arthritis (JIA) • Psoriatic arthritis • Ankylosing spondylitis • Crohn’s disease • Plaque psoriasis |
Labeled pediatric use(s) • Patients 4 to 17 yr for JIA • Safety and efficacy not established for children weighing <15 kg and for conditions other than JIA Study information • Safety and efficacy were assessed in patients 4 to 17 yr (n = 171). • Findings showed fewer patients in the adalimumab group than in placebo group experienced disease flare, regardless of methotrexate use. • Malignancies have been reported in children and adolescent patients receiving treatment with tumor necrosis factor blockers, of which adalimumab is a member. • Injection site reactions and infections are common adverse events. |
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4. Agalsidase beta |
Fabry disease |
Labeled pediatric use(s) • Patients 8 to 16 yr with Fabry disease • Safety and efficacy have not been evaluated in children <8 yr. Study information • Safety, pharmacokinetics, and pharmacodynamics were assessed in patients 8 to 16 yr (n = 16). • Ten of 12 patients taking agalsidase beta had a reduction in globotriaosylceramide to normal levels. • No new safety concerns were identified in pediatric patients. • Infusion reactions were the most common adverse event. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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5. Albumin (human) |
• Hypovolemia • Hypoalbuminemia • Prevention of central volume depletion after paracentesis • Ovarian hyperstimulation syndrome (25% only) • Adult respiratory distress syndrome (25%) • Acute nephrosis (25%) • Hemolytic disease of the newborn (25%) |
Labeled pediatric use(s) • Hypovolemia • Hemolytic disease of the newborn (25%) The product should only be administered to pediatric patients if needed. Study information • Data on use of albumin in children, including premature babies, are very limited. |
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6. Alefacept |
Chronic plaque psoriasis in adult patients | Safety and efficacy of Amevive in pediatric patients have not been studied. | ||
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7. Alemtuzumab |
B-cell chronic lymphocytic leukemia | Safety and effectiveness in pediatric patients have not been established. | ||
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8. Alglucosidase alfa |
Late-onset Pompe disease (α-glucosidase deficiency) |
Labeled pediatric use(s) • Patients >8 yr with late-onset Pompe disease • Safety and efficacy in pediatric patients <8 yrs have not been evaluated in clinical trials. Study information • Safety and efficacy were assessed in 90 patients with late-onset Pompe disease, ages 10 to 70 years, in a randomized double-blind, placebo-controlled study designed to enroll patients age 8-70 years. The youngest Lumizyme-treated patient was 16 years of age, and the youngest placebo-treated patient was 10 years of age. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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9. Alglucosidase alfa |
Pompe disease (α-glucosidase deficiency) |
Labeled pediatric use(s) • Infantile-onset Pompe disease (improvement in ventilator-free survival) • Risks and benefits have not been established in the juvenile-onset Pompe disease population. Study information • One trial assessed efficacy in patients ≤7 mo with infantile-onset Pompe disease (n = 18). A greater survival without invasive ventilator support was seen in patients receiving alglucosidase alfa vs. historical control. • A second trial assessed efficacy in patients 3 mo to 3.5 yr (n = 21). No effect of alglucosidase alfa compared with historical control could be determined. • Most common adverse reactions were infusion related. • Anaphylactic reactions, cardiorespiratory failure, and cardiac arrest have also occurred. • Open-label clinical trials have been performed in older pediatric patients ranging from 2 to 16 years at the initiation of treatment juvenile-onset Pompe disease). |
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10. Alpha1-proteinase |
Congenital deficiency of α1-proteinase inhibitor with clinically evident emphysema | Safety and effectiveness in pediatric patients have not been established. | ||
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11. Alpha1-proteinase |
Emphysema due to congenital deficiency of α1-proteinase inhibitor | Safety and effectiveness in pediatric patients have not been established. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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12. Alpha1-proteinase |
Congenital deficiency of α1-proteinase inhibitor with clinically evident emphysema | Safety and effectiveness in pediatric patients have not been established. | ||
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13. Anakinra |
Rheumatoid arthritis in adults |
Not recommended because prefilled syringes do not allow accurate dosing below 100 mg and efficacy could not be demonstrated in study because of low enrollment Study information • Efficacy was assessed in patients 2 to 17 yr (n = 86) with juvenile rheumatoid arthritis. • Efficacy was not demonstrated. An adverse event profile similar to that seen in adult patients with rheumatoid arthritis was observed. |
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14. Antihemophilic factor (recombinant) |
• Control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A • Short-term prophylaxis of spontaneous bleeding episodes in patients with hemophilia A |
Labeled pediatric use(s) • Appropriate for use in children of all ages with hemophilia A, including newborns Study information • Safety and efficacy studies have been performed with previously untreated neonates, infants, and children <1 to 52 mo (n = 101). • Studies were also performed with previously treated children and adolescents 5 to 18 yr (n = 31) |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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15. Antihemophilic factor (recombinant), plasma/albumin-free method |
• Control and prevention of bleeding episodes in adults and children with hemophilia A • Perioperative management in adults and children with hemophilia A |
Labeled pediatric use(s) • Control and prevention of bleeding episodes in adults and children with hemophilia A • Perioperative management in adults and children with hemophilia A Study information • Pharmacokinetic studies were performed in patients 1 mo to <16 yr (n = 51). • In comparison with adults, children had higher Factor VIII clearance values and thus lower half-lives and recovery of Factor VIII. • Larger or more frequent doses should be considered in a pediatric patient population. |
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16. Antihemophilic factor (recombinant), plasma/albumin free (Xyntha) |
• Control and prevention of bleeding episodes in patients with hemophilia A • Surgical prophylaxis in patients with hemophilia A |
Labeled pediatric use(s) • Bleeding episodes in hemophilia A • Surgical prophylaxis in hemophilia A • Description of indicated uses does not mention pediatric population explicitly. Study information • Pharmacokinetics were studied in previously treated patients 12 to 16 yr (n = 7). • Pharmacokinetic parameters were similar to those observed in adults. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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17. Antithrombin |
• Prevention of perioperative and peripartum thromboembolic events in hereditary antithrombin-deficient patients • Not indicated for treatment of thromboembolic events in hereditary antithrombin-deficient patients |
Safety and effectiveness in pediatric patients have not been established. | ||
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18. Anti-thymocyte globulin |
Acute rejection in renal transplant patients |
Safety and effectiveness in pediatric patients have not been established in controlled trials. Study information • Dose, efficacy, and adverse event profile are thought to be similar to those in adults, based on limited European studies and U.S. compassionate use. |
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19. Autologous cultured chondrocytes |
Repair of symptomatic cartilage defects of the femoral condyle | Safety and effectiveness in pediatric patients have not been established. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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20. Basiliximab |
Prophylaxis of acute organ rejection in patients receiving renal transplantation |
Labeled pediatric use(s) • Prophylaxis of acute organ rejection in renal transplant • In pediatric patients weighing less than 35 kg, the recommended regimen is two doses of 10 mg each. In pediatric patients weighing 35 kg or more, the recommended regimen is two doses of 20 mg each. Study information • Safety and pharmacokinetics were assessed in 39 pediatric patients. In patients 1 to 11 years of age (n = 25), disposition parameters were not influenced to a clinically relevant extent by age, body weight, or body surface area. In adolescents (12 to 16 years of age, n = 14), disposition was similar to that in adult renal transplantation patients. No randomized, controlled trials have been conducted in pediatric patients. • The adverse event profile was consistent with general clinical experience in pediatric renal transplantation population and with the profile in controlled adult renal transplantation studies. |
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21. Becaplermin |
Lower-extremity diabetic neuropathic ulcers | Safety and effectiveness in pediatric patients below 16 yr have not been established. | ||
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22. Bevacizumab |
• Metastatic colorectal cancer • Nonsquamous non-small-cell lung cancer • Metastatic breast cancer • Glioblastoma • Metastatic renal cell carcinoma |
Safety, effectiveness, and pharmacokinetic profile in pediatric patients have not been established. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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23. Botulism immune globulin intravenous |
Treatment of infant botulism by toxin types A or B in patients <1 yr |
Labeled pediatric use(s) • Treatment of infant botulism by toxin type A or B in patients <1 yr • BabyBIG has not been tested in other populations. Study information • Efficacy and safety were assessed in an infant population (n = 129). • BabyBIG was shown to significantly reduce hospital and intensive care unit stays, mechanical ventilation, and tube feeding. • The only noted adverse event was a mild rash on the face or trunk. |
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24. C1 esterase inhibitor (human) |
Treatment of acute abdominal or facial attacks of hereditary angioedema (HAE) in adult and adolescent patients |
Labeled pediatric use(s) • Treatment of attacks of HAE in patients >12 yr • Safety and efficacy in patients 0 to 12 yr have not been established. Study information • Pharmacokinetics and safety were assessed in patients 3 to 12 yr (n = 5) but numbers were insufficient to assess efficacy. |
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25. C1 esterase inhibitor |
Routine prophylaxis against angioedema attacks in adolescent and adult patients with hereditary angioedema (HAE) |
Labeled pediatric use(s) • Prophylaxis of attacks of HAE in adolescent and adult patients • Safety and effectiveness in neonates, infants, or children have not been established. Study information • Three patients <18 yr were included in a routine prophylaxis trial that found Cinryze to be effective in reducing days of swelling and mean severity and duration of attacks. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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26. Canakinumab |
Cryopyrin-associated periodic syndromes (CAPS) in adults and children ≥4 yr: • Familial cold autoinflammatory syndrome • Muckle-Wells syndrome |
Labeled pediatric use(s) • CAPS in patients ≥4 yr • Safety and effectiveness of Ilaris in patients under 4 yr have not been established. Study information • Safety and efficacy were assessed in the CAPS trial in patients 4 to 17 yr (n = 23). • The majority of patients achieved improvement in clinical symptoms and objective markers of inflammation. • Overall safety and efficacy of canakinumab in pediatric patients were comparable to those in adults. |
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27. Certolizumab pegol |
• Crohn’s disease • Rheumatoid arthritis |
Safety and effectiveness in pediatric patients have not been established. Boxed warning • Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with tumor necrosis factor blockers, of which certolizumab is a member. Certolizumab is not indicated for use in pediatric patients. |
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28. Cetuximab |
• Squamous cell carcinoma of the head and neck • Metastatic colorectal cancer |
Safety and effectiveness in pediatric patients have not been established. Study information • Pharmacokinetics were assessed in patients 1 to 12 yr (n = 27) and 13 to 18 yr (n = 19). • Pharmacokinetic profiles were similar between the age groups. • No new safety signals were identified in pediatric patients. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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29. Coagulation factor VIIa (recombinant) |
• Treatment of bleeding episodes in patients with hemophilia A or B and in acquired hemophilia • Prevention of bleeding in surgical interventions or invasive procedures in patients with hemophilia • Treatment of bleeding episodes in patients with congenital factor VII deficiency • Prevention of bleeding in surgical interventions or invasive procedures in patients with factor VII deficiency |
Labeled pediatric use(s) • All approved indications without explicit distinction by age Study information • Clinical trials enrolling pediatric patients were conducted, with dosing determined according to body weight and not according to age. • The safety and effectiveness of NovoSeven [for the indicated uses] has not been studied to determine if there are differences in various age groups, from infants to adolescents (0 to 16 yr) |
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30. Coagulation factor IX (recombinant) |
• Control and prevention of bleeding episodes in adult and pediatric patients with hemophilia • Perioperative management in adult and pediatric patients with hemophilia |
Labeled pediatric use(s) • Control and prevention of bleeding episodes in pediatric patients with hemophilia • Perioperative management in pediatric patients with hemophilia Study information • Pharmacokinetics, safety, and efficacy have been assessed in pediatric patients. • A lower recovery is generally observed for patients <15 yr; a dose adjustment may be needed. |
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31. Collagenase clostridium histolyticum |
Adult patients with Dupuytren’s contracture | Safety and effectiveness in pediatric patients have not been established. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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32. Crotalidae polyvalent immune Fab (ovine) |
Management of patients with North American crotalid (venomous snakes) envenomation |
Labeled pediatric use(s) • Specific studies with pediatric patients have not been conducted. The absolute venom dose following snakebite is expected to be the same in children and adults; therefore, no dosage adjustment for age should be made. Study information • Two clinical trials using CroFab have been conducted. They were prospectively defined, open-label, multicenter trials conducted with otherwise healthy patients 11 yr or older who had suffered from minimal or moderate North American crotalid envenomation that showed evidence of progression. |
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33. Daclizumab |
Prophylaxis of acute organ rejection in patients receiving renal transplants |
Labeled pediatric use(s) • Prophylaxis of acute organ rejection in patients 11 mo to 17 yr receiving renal transplants Study information • Pharmacokinetics, efficacy, immunogenicity, and safety were assessed in patients 11 mo to 17 yr (n = 60). • Patient and graft survival at 1 yr posttransplant were 100% and 96.7%, respectively. • Incidence of antidaclizumab antibodies (34%) was higher than incidence previously observed in adult patients. • Safety profile in pediatric population was comparable to that in adult patients, with some exceptions. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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34. Darbepoetin alfa (Aranesp) |
Treatment of anemia due to chronic kidney disease (CKD) or myelosuppressive chemotherapy |
Labeled pediatric use(s) • Treatment of anemia due to CKD in patients >1 yr and currently being treated with epoeitin alfa • Safety and efficacy have not been assessed in initial treatment of anemic pediatric patients with CKD. • Safety and efficacy in pediatric cancer patients have not been established. Study information • Pharmacokinetics were assessed in patients 3 to 16 yr (n = 12). • Pharmacokinetic parameters were similar to those obtained in adult patients. • Safety and the ability of darbepoetin to maintain hemoglobin concentrations in patients who had been receiving other recombinant erythropoietins were assessed in patients 1 to 17 yr (n = 123). • Efficacy and safety in the pediatric population were similar to those in the adult population. |
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35. Denileukin diftitox (Ontak) |
Cutaneous T-cell lymphoma | Safety and efficacy in pediatric patients have not been established. | ||
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36. Denosumab (Prolia/Xgeva) |
Xgeva: prevention of skeleton-related events in patients with bone metastases from solid tumors Prolia: treatment of postmenopausal women with osteoporosis |
Safety and effectiveness of denosumab in pediatric patients have not been established. Its use is not recommended in pediatric patients, as it may impair bone growth and may inhibit eruption of dentition. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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37. Digoxin immune Fab (ovine) |
Treatment of patients with life-threatening or potentially life-threatening digoxin toxicity or overdose |
Labeled pediatric use(s) Treatment of patients with life-threatening or potentially life-threatening digoxin toxicity or overdose including • known suicidal or accidental consumption of fatal doses of digoxin in adults or children; • chronic ingestion in adults and children; • manifestations of life-threatening toxicity due to digoxin overdose Study information No pediatric patients were enrolled in clinical studies of DigiFab. A similar digoxin ovine Fab product, Digibind, has been used successfully to treat infants. (Note: 2012 labeling states that safety data in pediatric population are limited and that pediatric dosing estimation is based on adult dose calculations.) |
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38. Drotrecogin alfa |
Reduction of mortality in adult patients with severe sepsis who have a high risk of death |
A placebo-controlled trial in pediatric patients did not establish safety and effectiveness. Study information • Safety and efficacy were assessed in pediatric patients in the RESOLVE trial (n = 477) • Study was terminated after interim analysis showed product was unlikely to show improvement over placebo. • Central nervous system bleeding was greater in drotrecogin alfa-treated patients than placebo. • All-cause mortality was similar in both treatment and placebo groups. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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39. Ecallantide |
Acute attacks of hereditary angioedema (HAE) | Safety and effectiveness of Kalbitor in patients <16 yr have not been established. | ||
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40. Eculizumab |
• Paroxysmal nocturnal hemoglobinuria to reduce hemolysis (PNH) |
Safety and effectiveness in pediatric patients <18 yr have not been established. (Note: FDA approved a new indication, atypical hemolytic uremic syndrome, in September 2011 with safety and effectiveness noted to be similar to adult patients based on studies that included 25 pediatric patients ages 2 months to 17 years.) |
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41. Etanercept |
• Rheumatoid arthritis (RA) • Polyarticular juvenile idiopathic arthritis (JIA) in patients >2 yr • Psoriatic arthritis • Ankylosing spondylitis • Plaque psoriasis |
Labeled pediatric use(s) • Polyarticular JIA in patients >2 yr • Not established for JIA in patients <2 yr • Safety and efficacy for plaque psoriasis in pediatric patients have not been established. Study information • Safety and efficacy were assessed in patients 2 to 17 yr with JIA (n = 69). • Significantly fewer patients who remained on etanercept than those who were on placebo experienced disease flare. Boxed warning • Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with tumor necrosis factor blockers, including etanercept. |
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42. Fibrin sealant |
Adjunct to hemostasis for use in cardiovascular surgery | Safety and effectiveness in pediatric patients undergoing cardiovascular surgery have not been established. | ||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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43. Fibrin sealant |
Adjunct to hemostasis in surgeries involving
• Cardiopulmonary bypass • Treatment of splenic injuries |
Safety and effectiveness in pediatric patients have not been established. (Note: In January 2012, FDA approved an indication extension for use as a general adjunct to hemostasis. The labeling notes that limited clinical data are available about use with children and notes that trial data on 27 revealed no differences in safety compared to the overall population studied.) |
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44. Fibrin sealant (human) |
To adhere autologous skin grafts to surgically prepared wound beds resulting from burns in adult and pediatric populations |
Labeled pediatric use(s) • Adherence of skin grafts to burns in patients ≥1 yr Study information • Safety and efficacy were assessed in patients 1 to 16 yr (n = 36). • Safety and efficacy did not differ from those in an adult population. |
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45. Fibrin sealant (human) |
Adjunct to hemostasis for use in patients during surgery |
Labeled pediatric use(s) • Adjunct to hemostasis for use in patients >6 mo during surgery Study information • No data were available for patients 0 to 6 mo. • Four pediatric patients were included in a study assessing use during retroperitoneal and intra-abdominal surgery; eight pediatric patients were included in a study assessing use during liver surgery. • On the basis of these data, use of Evicel in a pediatric population is supported. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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46. Fibrinogen concentrate (human) |
Treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency |
Statement of indicated use does not explicitly refer to pediatric patients Study information • Studies included patients <16 yr. • Patients <16 yr had shorter half-lives and faster clearance than adults. • Small numbers of subjects limit interpretation. |
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47. Galsulfase |
Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome) |
Labeled pediatric use(s) • MPS VI in patients ≥5 yr • Safety and efficacy in patients <5 yr have not been established. Study information • Clinical studies have been performed with patients 5 to 29 yr (n = 56). • Findings showed galsulfase to be effective at improving endurance in comparison with placebo. • An open-label study was conducted with four infants. • Safety results are consistent with results for patients 5 to 29 yr. |
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48. Golimumab |
• Rheumatoid arthritis • Psoriatic arthritis • Ankylosing spondylitis |
Safety and effectiveness of golimumab in pediatric patients <18 yr have not been established. Boxed warning • Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with tumor necrosis factor blockers, of which golimumab is a member. |
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
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49. Hepatitis B immune globulin intravenous (human) |
• Prevention of hepatitis B following liver transplantation • Postexposure prophylaxis in the following settings: acute exposure to blood containing hepatitis B virus (HBV) surface antigen (HBsAg), perinatal exposure of infants born to HBsAg-positive mothers, sexual exposure to HBsAg-positive persons, household exposure to persons with acute HBV infection |
Labeled pediatric use(s) • Perinatal exposure of infants born to HBsAg-positive mothers • Safety and effectiveness have not been established in pediatric patients. However, for postexposure prophylaxis, the safety and effectiveness of similar hepatitis B immune globulins have been demonstrated in infants and children. |
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|
||||
50. Hepatitis B immune globulin (human) |
Treatment of • Acute exposure to blood containing hepatitis B virus (HBV) surface antigen (HBsAg) • Perinatal exposure of infants born to HBsAg-positive mothers • Sexual exposure to HBsAg-positive persons • Household exposure to persons with acute HBV infection |
Labeled pediatric use(s) • Perinatal exposure of infants born to HBsAg-positive mothers • Infants less than 12 mo old whose mother or primary caregiver is positive for HBsAg Study information • Safety and efficacy in the pediatric population have not been established. However, safety and effectiveness of similar hepatitis B immune globulins have been demonstrated in infants and children. |
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|
||||
51. Ibritumomab tiuxetan |
• Low-grade B-cell non-Hodgkin’s lymphoma (NHL) • Follicular NHL |
Safety and effectiveness of Zevalin in pediatric patients have not been established. |
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|
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
52. Idursulfase (Elaprase) |
Hunter syndrome (Mucopolysaccharidosis II [MPS II]) |
Labeled pediatric use(s) • Hunter syndrome in patients ≥5 yr • Safety and efficacy in pediatric patients <5 yr have not been established. Study information • Safety and efficacy have been evaluated in patients 5 to 31 yr (n = 96). • Findings showed improved walking capacity in patients receiving idursulfase compared with that in patients receiving placebo. • Children, adolescents, and adults responded similarly to treatment with idursulfase. • Adverse effects include infusion-related reactions and hypoxemic episodes. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
53. Immune globulin injection (human), 10%, caprylate/chromatography purified (Gamunex-C) |
• Primary humoral immunodeficiency (PI) • Idiopathic thrombocytopenic purpura (ITP) • Chronic inflammatory demyelinating polyneuropathy (CIDP) |
Labeled pediatric use(s) • PI (intravenous route) in pediatric patients • ITP (intravenous route) in pediatric patients • Efficacy and safety for CIDP in pediatric patients have not been established. • Efficacy and safety by the subcutaneous route in pediatric patients have not been established. Study information • Intravenous Gamunex-C was evaluated for treatment of PI in pediatric patients 0 to 16 yr (n = 18). • Pharmacokinetics, safety, and efficacy were similar to those in an adult population. • Vomiting was more frequent in the pediatric population. • Subcutaneous Gamunex-C was evaluated in three pediatric patients with PI; this number was too small to evaluate them separately from an adult population. • Intravenous Gamunex-C was evaluated for the treatment of ITP in pediatric patients (n = 12). • Pharmacokinetics, safety, and efficacy were similar to those in an adult population. • Fever was more frequent in the pediatric population. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
54. Immune globulin intravenous (human) |
Treatment of primary humoral immunodeficiency disorders |
Efficacy and safety in pediatric patients have not been established. Study information |
||
|
||||
55. Immune globulin intravenous (human), 5% liquid |
Treatment of primary humoral immunodeficiency diseases |
Labeled pediatric use(s) • Primary humoral immunodeficiency in patients ≥6 yr (implicit) Study information • Pediatric patients 6 to 16 yr were included in a clinical study (n = 11). • Pharmacokinetics, safety, and efficacy were similar to those in an adult population. No pediatric-specific dose requirements were necessary to achieve the desired serum IgG levels. |
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|
||||
56. Immune globulin intravenous (human), 10% solution |
Treatment of primary humoral immunodeficiency |
Labeled pediatric use(s) • Primary humoral immunodeficiency in patients ≥2 yr • Safety and efficacy have not been established in patients <2 yr. Study information • Safety and efficacy were evaluated in well-controlled studies that included pediatric patients 2 to 16 yr. • Results were similar to those seen in adults. No adjustments in dosing were necessary. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
57. Immune globulin intravenous (human), 10% liquid |
• Primary humoral immunodeficiency • Chronic immune thrombocytopenic purpura |
Labeled pediatric use(s) • Primary humoral immunodeficiency in patients ≥3 yr • Safety and effectiveness of Privigen have not been established in pediatric patients <3 yr. • Safety and effectiveness of Privigen have not been established in pediatric patients <15 yr with chronic immune thrombocytopenic purpura. Study information • Safety and efficacy were assessed in pediatric patients with PI (n = 31). • Safety and efficacy profiles were comparable to those for adults. • Safety, efficacy, and tolerability were established in patients 15 to 69 yr with ITP. |
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|
||||
58. Immune globulin subcutaneous (human) |
Treatment of patients with primary humoral immunodeficiency |
Labeled pediatric use(s) • Treatment of primary humoral immunodeficiency • Safety and efficacy in pediatric subjects <2 yr have not been established. Study information • Two studies enrolled pediatric patients 3 to 16 yr (n = 10, n = 22). • Safety and efficacy were similar to those seen in an adult population. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
59. Immune globulin subcutaneous (human) (IGSC), 20% liquid (Hizentra) |
Treatment of primary immunodeficiency |
Labeled pediatric use(s) • Primary immunodeficiency in patients 2 to 16 yr • Safety and efficacy in pediatric patients <2 yr have not been established. Study information • Safety and efficacy have been established in a U.S. study (n = 10) and a European study (n = 23). • Safety and efficacy profiles were similar to those for an adult population. |
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|
||||
60. Immune globulin intravenous (human), 5% liquid |
Primary humoral immunodeficiency |
Implicit that safety and efficacy in a pediatric population have not been established. Study information • Six pediatric patients were included in a study but could not be evaluated separately because of small sample size. |
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|
||||
61. IncobotulinumtoxinA |
• Cervical dystonia • Blepharospasm |
Safety and efficacy of incobotulinumtoxinA in patients <18 yr have not been established. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
62. Infliximab (Remicade) |
• Crohn’s disease (CD) • Ulcerative colitis • Ankylosing spondylitis • Psoriatic arthritis • Plaque psoriasis |
Labeled pediatric use(s) • CD in patients ≥6 yr • Use not established for patients <6 yr with CD • Long-term safety and efficacy in pediatric CD patients not determined • Use by pediatric patients with ulcerative colitis and plaque psoriasis not established Study information • Safety and efficacy have been assessed in patients 6 to 17 yr with CD (n = 112). • Findings showed that infliximab was effective at reducing CD signs and symptoms and maintaining clinical remission. • Safety and efficacy were assessed in patients 4 to 17 yr with juvenile rheumatoid arthritis (n = 60). • Study failed to establish efficacy of infliximab in patients with juvenile rheumatoid arthritis. • Findings showed high placebo response rate and higher rate of immunogenicity in pediatric patients than in adults. Boxed warning • Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with tumor necrosis factor (TNF) blockers, of which infliximab is a member. • Postmarketing cases of hepatosplenic T-cell lymphoma have been reported in patients treated with TNF blockers, including infliximab. All cases were reported in patients with Crohn’s disease and ulcerative colitis, the majority of whom were adolescent or young adult males. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
63. Interferon alfacon-1 |
Chronic hepatitis C virus infection in patients ≥18 yr | Safety and effectiveness of interferon alfacon-1 in patients <18 yr have not been established. It is not recommended as therapy in pediatric patients. | ||
|
||||
64. Interferon beta-1A (Rebif) |
Relapsing forms of multiple sclerosis | Safety and effectiveness of interferon beta-1A in pediatric patients have not been studied. | ||
|
||||
65. Interferon gamma-1B (Actimmune) |
• Reduction in infections in patients with chronic granulomatous disease (CGD) • Delaying disease progression of osteopetrosis |
Labeled pediatric use(s) • CGD • Osteopetrosis • Statements of indicated uses do not explicitly refer to pediatric patients. Study information • Safety and efficacy were assessed in patients 1 to 44 yr with CGD (median age 14.6 yr) (n = 128). • A statistically significant benefit in time to serious infection was found in the interferon gamma-1B group compared with placebo group. • Safety and efficacy were assessed in patients 1 mo to 8 yr with osteopetrosis (n = 16). • Median time to disease progression was delayed in the group receiving interferon gamma-1B plus calcitriol vs. the group receiving calcitriol alone. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
66. Laronidase (Aldurazyme) |
Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I) |
Labeled pediatric use(s) • Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie form who have moderate to severe symptoms Study information • Two studies assessed safety and efficacy in patients 6 to 43 yr (n = 45). • Improvement in breathing and walking capacities were found in the laronidase group compared with placebo group. • A third study assessed safety and efficacy in patients 6 mo to 5 yr (n = 20). • Safety and efficacy findings were similar to those from a study that included both pediatric and adult populations. • Common adverse events included infusion reactions and otitis media. |
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|
||||
67. Natalizumab |
• Relapsing forms of multiple sclerosis (MS) • Crohn’s disease (CD) |
Safety and effectiveness of Tysabri in pediatric patients <18 yr with MS or CD have not been established. Tysabri is not indicated for use by pediatric patients. |
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|
||||
68. Ofatumumab |
Chronic lymphocytic leukemia | Safety and effectiveness in pediatric patients have not been established. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
69. Omalizumab |
Moderate to severe asthma |
Labeled pediatric use(s) • Moderate to severe persistent asthma in patients ≥12 yr • Not indicated for patients <12 yr Study information • Safety and effectiveness were assessed in two studies with asthma patients 6 to <12 yr (n = 926). • Exacerbations were reduced, but other efficacy measures did not differ from those for placebo group. • Known risk of anaphylaxis and malignancy in patients ≥12 yr outweighs benefit in children <12 yr. |
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|
||||
70. Oprelvekin |
Prevention of severe thrombocytopenia following myelosuppressive chemotherapy |
A safe and effective dose of Neumega in pediatric patients has not been established. Study information • A dose-escalation study involving 43 pediatric patients did not reduce need for transfusions and projected the effective dose to be higher than the maximum tolerated pediatric dose. • Papilledema was a dose-limiting adverse effect. |
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|
||||
71. Palifermin |
Oral mucositis | Safety and effectiveness of Kepivance in pediatric patients have not been established. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
72. Palivizumab |
Prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in pediatric patients |
Labeled pediatric use(s) • Prevention of lower respiratory tract disease caused by RSV in pediatric patients Study information • Safety and efficacy were assessed in two studies with patients ≤24 mo (n = 2,789). • Findings showed a significant reduction in hospitalization for RSV infection in patients receiving palivizumab than those receiving placebo. |
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|
||||
73. Panitumumab |
Metastatic colorectal carcinoma | Pharmacokinetics, safety, and effectiveness in pediatric patients have not been established. | ||
|
||||
74. Pegfilgrastim |
To decrease infections in patients receiving myelosuppressive anticancer drugs associated with febrile neutropenia |
Safety and efficacy of Neulasta in pediatric patients have not been established. Study information • Pharmacokinetics and safety studies were conducted with 37 pediatric patients with sarcoma. • The most common adverse reaction was bone pain. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
75. Peginterferon alfa-2A |
Chronic hepatitis C (CHC) |
Labeled pediatric use(s) • CHC in patients 5 yr and older with compensated liver disease not previously treated with interferon alpha and patients with histological evidence of cirrhosis and compensated liver disease was treated with Peginterferon alfa-2A. Peginterferon alfa-2A should be given in combination with Copegus unless contraindicated. Peginterferon alfa-2A contains benzyl alcohol, which has been associated with an increased incidence of neurological and other complications in neonates and infants. Study information • Information on safety, dosing, and efficacy from a randomized trial (114 subjects) comparing combination with monotherapy is available. • Pediatric subjects treated with Pegasys plus Copegus combination therapy showed delays in weight and height increases after 48 wk of therapy compared with those at baseline. |
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|
||||
76. Peginterferon alfa-2A; ribavirin |
Chronic hepatitis C | See labeling information for Pegasys. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
77. Peginterferon alfa-2B |
Combination therapy • In combination with ribavirin for chronic hepatitis C in patients ≥3 yr Monotherapy • Chronic hepatitis C in patients ≥18 yr |
Labeled pediatric use(s) • In combination with ribavirin for chronic hepatitis C in patients ≥3 yr • Safety and effectiveness of peginterferon alfa-2B in combination with ribavirin in pediatric patients <3 yr have not been established. Study information • Safety and efficacy of peginterferon alfa-2B and ribavirin were established in patients 3 to 17 yr (n = 107) |
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|
||||
78. Pegloticase |
Chronic gout in adult patients | Safety and efficacy in pediatric patients <18 yr have not been established. | ||
|
||||
79. Protein C concentrate (human) |
Prevention and treatment of venous thrombosis and purpura fulminans (PF) in congenital protein C deficiency |
Labeled pediatric use(s) • Recommended for neonate and pediatric use Study information • Several retrospective and prospective studies have evaluated safety and efficacy in neonates and pediatric patients. • A pivotal study assessed the efficacy of Ceprotin in treating PF and other thromboembolic events in patients 0 to 25 yr (n = 18). • When compared with a historical control group, Ceprotin was more effective than fresh frozen plasma or other conventional anticoagulants. |
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|
||||
80. Ranibizumab |
• Macular degeneration • Macular edema |
Safety and efficacy in pediatric patients have not been established. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
81. Rasburicase |
Management of hyperuricemia in patients with leukemia, lymphoma, and solid tumor malignancies who are receiving anticancer therapy expected to result in tumor lysis and subsequent elevation of plasma uric acid |
Labeled pediatric use(s) • Hyperuricemia in pediatric patients with malignancies who are receiving anticancer therapy expected to result in tumor lysis Study information • Safety and efficacy in patients 1 mo to 17 yr were studied (n = 246). • Children <2 yr had a lower rate of achieving normal uric acid concentrations than those 2 to 17 yr. • Incidence of renal failure was similar between the rasburicase and allopurinol groups. |
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|
||||
82. Rho(D) immune globulin intravenous (human) |
• Suppression of Rhesus (Rh) isoimmunization in – pregnancy and obstetric conditions – Incompatible transfusions in Rho (D)-negative individuals • raising platelet counts in adults with idiopathic thrombocytopenic purpura |
• Safety and effectiveness in pediatric subjects being treated for an incompatible transfusion have not been established. • The physician should weigh the potential risks against the benefits of Rhophylac, particularly in girls whose later pregnancies may be affected if Rh isoimmunization occurs. |
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
83. Rilonacept |
Cryopyrin-associated periodic syndromes (CAPS) |
Labeled pediatric use(s) • CAPS, including familial cold autoinflammatory syndrome and Muckle-Wells syndrome in patients ≥12 yr • Safety and efficacy in patients <12 yr have not been established. Study information • Pharmacokinetics, safety, and efficacy in patients 12 to 16 yr were assessed (n = 6). • Findings showed improvement in baseline symptom scores and in markers of inflammation. • It is unknown whether rilonacept will alter bone development in children. |
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|
||||
84. Rimabotulinum-toxinB |
Cervical dystonia | Safety and effectiveness in pediatric patients have not been established. | ||
|
||||
85. Rituximab |
• Non-Hodgkin’s lymphoma • Chronic lymphocytic leukemia • Rheumatoid arthritis • Wegener’s granulomatosis and microscopic polyangiitis |
• The safety and effectiveness of Rituxan in pediatric patients have not been established. • FDA has not required pediatric studies of patients 0 to 16 yr with polyarticular juvenile idiopathic arthritis because of concerns regarding the potential for prolonged immunosuppression. |
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|
||||
86. Romiplostim |
Chronic immune (idiopathic) thrombocytopenic purpura | Safety and effectiveness in pediatric patients <18 yr have not been established. | ||
|
||||
87. Sipuleucel T |
Metastatic hormone-refractory prostate cancer | No pediatric use section or other reference to children in label. By implication, safety and effectiveness not established in pediatric patients. | ||
|
||||
88. Tenecteplase |
Reduction in mortality associated with acute myocardial infarction | Safety and effectiveness in pediatric patients have not been established. | ||
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|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
89. Thrombin, topical (human) |
Aid to hemostasis during surgery |
Labeled pediatric use(s) • Aid to hemostasis during surgery in pediatric patients Study information • Safety and efficacy were established in a clinical trial that included 8 pediatric patients 0 to 12 yr undergoing liver surgery. |
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|
||||
90. Thrombin, topical (recombinant) |
Aid to hemostasis during surgery |
Safety and effectiveness in a pediatric population have not been fully established. Study information • Recothrom was evaluated in four pediatric patients 12 to 16 yr. |
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|
||||
91. Tocilizumab (Actemra) |
• Rheumatoid arthritis • Systemic juvenile idiopathic arthritis (SJIA) |
Labeled pediatric use(s) • SJIA • Safety and effectiveness in pediatric patients with conditions other than SJIA have not been established. Study information • Efficacy and safety in pediatric patients with SJIA were assessed (n = 75). • The response in the Actemra group was significant compared with that in the placebo group. |
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|
||||
92. Tositumomab; iodine I 131 tositumomab |
Non-Hodgkin’s lymphoma | Safety and effectiveness of Bexxar in children have not been established. | ||
|
||||
93. Trastuzumab |
• HER2-overexpressing breast cancer • HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma |
Safety and effectiveness of Herceptin in pediatric patients have not been established. | ||
|
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Approved Indication(s) | Highlights of Pediatric Information in Labeling | ||
|
||||
94. Ustekinumab |
Plaque psoriasis in adult patients | Safety and effectiveness of Stelara in pediatric patients have not been evaluated. | ||
|
||||
95. Vaccinia immune globulin intravenous |
• Eczema vaccinatum • Progressive vaccinia • Severe generalized vaccinia • Vaccinia virus infections in patients with skin conditions |
Safety and effectiveness in the pediatric population (<16 yr) have not been established. | ||
|
||||
96. von Willebrand factor/coagulation factor VIII complex (human) |
Treatment of bleeding episodes in patients with von Willebrand disease (VWD) |
Labeled pediatric use(s) • Statement of indicated use does not explicitly refer to pediatric patients. Study information • Eleven pediatric patients between 5 and 16 yr with VWD (eight with type 3, one with type 2, two with type 1) were treated with Wilate for 234 bleeding episodes (BEs) in clinical studies. These studies showed that 88% of the BEs were successfully treated in this population. No dose adjustment is needed for pediatric patients, as administered dosages were similar to those used by the adult population. |
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|
TABLE D-2 Pediatric Trials Registered at ClinicalTrials.gov for Biologics Initially Approved by FDA Between January 1, 1997, and December 31, 2010 (listed separately for CDER-and CBER-regulated products)
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
1. | Abatacept (Orencia) (125118) 12/23/2005 |
Juvenile rheumatoid arthritis | 6 to 17 yr | III |
Type 1 diabetes mellitus | 6 to 45 yr | II | ||
Acute graft-versus-host disease during transplant | >12 yr | II | ||
Wegener’s granulomatosis | >15 yr | I/II | ||
Uveitis | >6 yr | II | ||
|
||||
2. | AbobotulinumtoxinA (Dysport) (125274) 04/29/2009 |
Spasticity in cerebral palsy | 2 to 17 yr | III |
Idiopathic toe walking | 5 to 15 yr | II | ||
Cerebral palsy | 25 mo to 9 yr | IV | ||
Leg length inequality; foot deformities | 6 to 16 yr | IV | ||
Torticollis | 4 mo to 1 yr | I | ||
Lower limb length discrepancy | 5 to 21 yr | III | ||
Myelomeningocele; neurogenic bladder | 2 to 16 yr | IV | ||
Cerebral palsy | 1 to 17 yr | I/II | ||
Muscle spasticity in cerebral palsy | 3 to 12 yr | II | ||
Spastic diplegic cerebral palsy | 3 to 18 yr | III | ||
Cerebral palsy | 4 to 12 yr | I/II | ||
Spasticity | >2 yr | III | ||
Cerebral palsy; drooling | 6 to 21 yr | n/s | ||
Cerebral palsy | 5 to 15 yr | n/s | ||
Idiopathic clubfoot | 1 day to 2 yr | n/s | ||
Spasticity in cerebral palsy | 2 to 18 yr | IV | ||
Clubfoot | Up to 12 yr | n/s | ||
Stroke; brain injuries; spasticity | >12 yr | IV | ||
Spinal cord injury; pain | >15 yr | n/s | ||
Cerebral palsy | 2 to 18 yr | I/II | ||
Spasticity, poststroke | >2 yr | n/s | ||
Hyperhidrosis | 12 to 17 yr | IV | ||
Cerebral palsy | 10 to 17 yr | II | ||
Esotropia | Up to 5 yr | n/s | ||
Hip pain in cerebral palsy | 4 to 16 yr | II | ||
Cerebral palsy | 8 to 11 yr | IV | ||
|
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
3. | Adalimumab (Humira) (125057) 12/31/2002 |
Juvenile rheumatoid arthritis | 4 to 17 yr | III |
Juvenile idiopathic arthritis | 4 to 17 yr | III | ||
Plaque psoriasis | 4 to 17 yr | III | ||
Juvenile idiopathic arthritis | 4 to 17 yr | n/s | ||
Juvenile idiopathic arthritis | 2 to 4 yr | III | ||
Enthesitis-related arthritis (ERA) | 6 to 17 yr | III | ||
Focal glomerulosclerosis | 2 to 40 yr | I | ||
Focal segmental glomerulosclerosis | 1 to 50 yr | II | ||
Uveitis; juvenile arthritis | >4 yr | II/III | ||
Crohn’s disease | 6 to 17 yr | III | ||
Crohn’s disease | 15 to 75 yr | II/III | ||
Intestinal Behcet’s disease | >15 yr | III | ||
Ankylosing spondylitis | >15 yr | III | ||
Ulcerative colitis | >15 yr | III | ||
Crohn’s disease | 7 to 18 yr | III | ||
Crohn’s disease | 15 to 75 yr | II/III | ||
Crohn’s disease-like inflammatory bowel disease in chronic granulomatous disease | >10 yr | I/II | ||
|
||||
4. | Agalsidase beta (Fabrazyme) (103979) 04/24/2003 |
Fabry disease | 7 to 15 yr | II |
Fabry disease | Infants | IV | ||
Fabry disease | 5 to 85 yr | IV | ||
Fabry disease | 5 to 18 yr | III | ||
Fabry disease | >15 yr | IV | ||
Fabry disease | 8 to 18 yr | n/s | ||
Fabry disease; proteinuria | 14 to 95 yr | n/s | ||
|
||||
5. | Alefacept (Amevive) (125036) 01/30/2003 |
Hematopoietic stem cell transplant | Up to 21 yr | n/s |
Type 1 diabetes mellitus | 12 to 35 yr | II | ||
Psoriasis | 12 to 17 yr | II | ||
Graft-versus-host disease | 14 to 75 yr | III | ||
Resistant chronic graft-versus-host disease | Up to 70 yr | I/II | ||
|
||||
6. | Alemtuzumab (Campath) (103948) 05/07/2001 |
In association with stem cell transplants for various hematologic malignancies, multiple trials | Various age ranges across pediatric population | 0, I, II, III |
Acute lymphoblastic leukemia | Up to 30 yr | II | ||
Aplastic anemia | ≥2 yr | II | ||
Chronic lymphocytic leukemia | Up to 69 yr | II | ||
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
7. | Alglucosidase alfa (Lumizyme) (125291) 05/24/2010 |
Pompe disease (late onset) Pompe disease (late onset) Pompe disease (infantile onset) Pompe disease (infantile onset) Pompe disease |
>8 yr >1 yr Up to 26 wk 6 to 36 mo >1 mo |
III n/s II/III I/II IV |
8. | Alglucosidase alfa (Myozyme) (125141) 04/28/2006 |
Pompe disease Pompe disease (late onset) Pompe disease Pompe disease Pompe disease (infantile onset) Pompe disease Pompe disease (late onset) |
<18 yr 8 to 18 yr >6 mo Up to 24 mo <12 mo >8 yr >8 yr |
IV IV IV IV n/s n/s IV |
9. | Anakinra (Kineret) (103950) 11/14/2001 |
Type 1 diabetes mellitus | 6 to 18 yr | I/II |
Atopic dermatitis | 10 to 18 yr | I | ||
Juvenile chronic arthritis | 2 to 17 yr | II | ||
Juvenile idiopathic arthritis | 2 to 20 yr | II/III | ||
Neonatal-onset multisystem inflammatory disease | Neonates | |||
Relapsing polychondritis | 12 to 15 yr | II | ||
10. | Basiliximab (Simulect) (103764) 05/12/1998 |
Liver transplantation complications | Up to 16 yr | IV |
Kidney transplantation complications | Up to 20 yr | n/s | ||
Noninfectious uveitis | 12 to 80 yr | II | ||
Kidney transplantation complications | 1 to 18 yr | III | ||
11. | Becaplermin (Regranex) (103691) 12/16/1997 |
|||
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
12. | Bevacizumab (Avastin) (125085) 02/26/2004 |
Solid tumors | Up to 21 yr | I |
Glial cell tumors | 3 to 21 yr | II | ||
Central nervous system tumors | 18 mo to 23 yr | I | ||
Intrinsic pontine glioma | 3 to 18 yr | II | ||
Central nervous system tumors | 1 to 25 yr | n/s | ||
Pulmonary vein stenosis | No age range given; infants and children | n/s | ||
Brain cancer | Up to 21 yr | II | ||
Gliomas | 3 to 30 yr | n/s | ||
Central nervous system tumors | 3 to 21 yr | II/III | ||
Neuroblastoma | Up to 30 yr | I | ||
Refractory solid tumors | 12 mo to 20 yr | I | ||
Medullablastoma | Up to 19 yr | II | ||
Central nervous system tumors | Up to 21 yr | II | ||
Sarcoma | Up to 29 yr | II | ||
Osteosarcoma | Up to 30 yr | III | ||
Solid tumor | 1 to 30 yr | I/II | ||
Central nervous system tumors | Up to 21 yr | II | ||
Refractory solid tumors; leukemia | Up to 21 yr | I | ||
Sarcoma | 6 mo to 18 yr | II | ||
Retinopathy of prematurity | 30 wk and older | n/s | ||
Neurofibromatosis type 2 | ≥12 yr | II | ||
Retinopathy of prematurity | 30 to 36 wk | I | ||
Sarcoma | 1 to 29 yr | II | ||
Refractory solid tumors | 1 to 21 yr | I | ||
Retinopathy of prematurity | Up to 22 wk | II | ||
Retinopathy of prematurity | 30 to 36 wk | II | ||
Retinopathy of prematurity | 1 to 12 mo | II/III | ||
Central nervous system tumors | ≥15 yr | I | ||
Sarcoma | ≥13 yr | II | ||
Neovascular glaucoma | 14 to 72 yr | II | ||
Sarcoma | 1 to 29 yr | n/s | ||
Neovascular glaucoma | 10 to 80 yr | n/s | ||
Glioma | 3 to 18 yr | II | ||
Gastrointestinal cancer | ≥18 mo | II | ||
Germ cell tumors | 12 to 65 yr | II | ||
Neuroblastoma | ≥1 yr | I | ||
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
13. | Canakinumab (Ilaris) (125319) 06/17/2009 |
Cryopyrin-associated periodic syndromes | ≥2 yr Up to 4 yr ≥3 yr 4 to 75 yr |
III (all) |
Systemic juvenile arthritis | ≥2 yr 2 to 19 yr |
III (all) | ||
Type 1 diabetes mellitus | 6 to 45 yr 6 to 35 yr |
II | ||
Familial Mediterranean fever | 4 to 20 yr 12 to 75 yr |
II | ||
Neonatal-onset multisystem inflammatory disease | 2 to 25 yr | III | ||
Mevalonate kinase deficiency | ≥2 yr | II | ||
4 to 75 yr | II | |||
Tumor necrosis factor receptor-associated periodic syndromes | ≥4 yr | II | ||
14. | Certolizumab pegol (Cimzia) (125160) 04/22/2008 |
Crohn’s disease (several) | Varying, 6 to 65 yr | II (all) |
Plaque psoriasis | Up to 18 yr | II | ||
15. | Cetuximab (Erbitux) (125084) 02/12/2004 |
Brain cancer | 3 to 21 yr | II |
Refractory solid tumors | 1 to 18 yr | I | ||
16. | Collagenase (Xiaflex) (125338) 02/02/2010 |
|||
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
17. | Daclizumab (Zenapax) (103749) 12/10/1997 |
Immune suppression in kidney transplantation | Up to 21 yr | |
Cardiac transplantation complications | 1 mo to 18 yr | I/II | ||
Cardiac transplantation complications | Up to 21 yr | n/s | ||
Juvenile idiopathic arthritis-associated uveitis | 6 to 18 yr | II | ||
Type I diabetes mellitus | 8 to 45 yr | III | ||
Type I diabetes mellitus | 2 to 40 yr | II | ||
Leukemia | ≥10 yr | II | ||
Anemia | ≥2 yr | II | ||
Ulcerative colitis | ≥12 yr | II | ||
Cardiac transplantation complications | ≥13 yr | IV | ||
Cystinosis | ≥7 yr | n/s | ||
Uveitis | ≥6 yr | n/s | ||
Uveitis | ≥13 yr | IV | ||
|
||||
18. | Darbepoetin alfa (Aranesp) (103951) 09/17/2001 |
Anemia due to chronic renal failure | Up to 17 yr | III |
Anemia of prematurity | Up to 49 h | II | ||
Anemia due to chronic kidney disease | 1 to 18 yr | III | ||
|
||||
19. | Denileukin diftitox (Ontak) (103767) 02/05/1999 |
Anaplastic large-cell lymphoma | 2 to 24 yr | II |
Graft-versus-host disease | ≥2 yr | II | ||
Neuroblastoma | Up to 21 yr | II | ||
Neuroblastoma | Up to 21 yr | I | ||
Graft-versus-host disease | ≥6 yr | II | ||
Refractory lymphoid malignancies | Any age | II | ||
Leukemia | ≥2 yr | II | ||
|
||||
20. | Denosumab (Prolia/Xgeva) (125320) 06/01/2010 |
Giant-cell tumor of bone | ≥12 yr | II |
|
||||
21. | Xigris (125029) 11/21/2001 |
Severe sepsis | Up to 17 yr | III |
|
||||
22. | Ecallantide (Kalbitor) (125277) 11/27/2009 |
Hereditary angioedema | ≥10 yr | n/s |
Hereditary angioedema | ≥10 yr | III | ||
Hereditary angioedema | 2 to 17 yr | II/III | ||
Hereditary angioedema | ≥10 yr | III | ||
|
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
23. | Eculizumab (Soliris) (125166) 03/16/2007 |
Hemoglobinuria | 2 to 17 yr | I/II |
Atypical hemolytic-uremic syndrome | 12 to 18 yr | II | ||
Atypical hemolytic-uremic syndrome | 12 to 18 yr | II | ||
Hemoglobinuria | ≥12 yr | II | ||
Hemoglobinuria | ≥12 yr | II | ||
Shiga toxin hemolytic-uremic syndrome | ≥2 mo | II/III | ||
Atypical hemolytic-uremic syndrome | 1 mo to 18 yr | II | ||
Hemolytic-uremic syndrome | ≥2 yr | n/s | ||
|
||||
24. | Etanercept (Enbrel) (103795) 11/02/1998 |
Polyarticular juvenile idiopathic arthritis | ≥2 mo | IV |
Fanconi anemia | ≥4 yr | n/s | ||
Kawasaki disease | 2 mo to 20 yr | II | ||
Psoriasis | 4 to 17 yr | n/s | ||
Psoriasis | 4 to 17 yr | III | ||
Idiopathic pneumonia syndrome after stem cell transplant | 1 to 17 yr | II | ||
Histiocytosis | Up to 65 yr | II | ||
Type 1 diabetes mellitus | 3 to 18 yr | I/II | ||
Graft-versus-host disease, multiple trials | Various age ranges across pediatric population | II, III | ||
Dermatomyositis | 4 to 16 yr | II/III | ||
Idiopathic pneumonia syndrome after stem cell transplant | ≥6 yr | II | ||
Wegener’s granulomatosis | 10 to 70 yr | II | ||
Psoriasis | n/s | III | ||
Leukemia | 2 to 18 yr | III | ||
Uveitis | Any age | II | ||
|
||||
25. | Galsulfase (Naglazyme) (125117) 05/31/2005 |
Mucopolysaccharidosis VI, multiple trials | Various age ranges | I, II, III, IV |
|
||||
26. | Golimumab (Simponi) (125289) 04/24/2009 |
Juvenile idiopathic arthritis | 2 to 18 yr | III |
|
||||
27. | Ibritumomab tiuxetan (Zevalin) (125019) 02/19/2002 |
Lymphoma | Up to 21 yr | I |
Non-Hodgkin’s lymphoma | Up to 64 yr | II | ||
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
28. | Idursulfase (Elaprase) (125151) 07/24/2006 |
Hunter syndrome | ≥5 yr | n/s |
Hunter syndrome | ≥5 yr | II/III | ||
Mucopolysaccharidosis II | 5 to 25 yr | II/III | ||
Hunter syndrome | 3 to 18 yr | n/s | ||
Mucopolysaccharidosis II | 6 to 35 yr | II/III | ||
29. | IncobotulinumtoxinA (Xeomin) (125360) 07/30/2010 |
|||
30. | Infliximab (Remicade) (103772) 08/24/1998 |
Juvenile rheumatoid arthritis Ulcerative colitis Ulcerative colitis Graft-versus-host disease Graft-versus-host disease Kawasaki disease Kawasaki disease Uveitis Juvenile idiopathic arthritis Juvenile idiopathic arthritis Juvenile idiopathic arthritis Spondylarthropathies Crohn’s disease Chronic granulomatous disease Uveitis |
4 to 18 yr 6 to 18 yr 6 to 17 yr 6 mo to 75 yr Up to 18 yr Up to 18 yr Up to 17 yr Up to 18 yr 4 to 15 yr 1 to 16 yr 4 to 18 yr Up to 18 yr 6 to 17 yr ≥10 yr ≥9 yr |
II n/s III II I I III IV III n/s III II/III III I/II n/s |
31. | Interferon alfacon-1 (Infergen) (103663) 10/06/1997 |
|||
32. | Interferon beta-1A (Rebif) (103780) 03/07/2002 |
Clinically isolated syndrome | 18 mo to 65 yr | III |
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
33. | Interferon gamma-1B (Actimmune) (103836) 02/25/1999 |
Osteopetrosis | 2 mo to 10 yr | III |
Chronic granulomatous disease | All ages | IV | ||
HIV infection | 1 to 17 yr | I | ||
Lymphoma | Up to 20 yr | II/III | ||
Leukocyte adhesion deficiency syndrome | Children, not specified | II | ||
Pulmonary tuberculosis | ≥5 yr | II | ||
Nontuberculosis mycobacterial infections | ≥5 yr | II | ||
Cystic fibrosis | ≥12 yr | I/II | ||
Fungal infections | ≥2 yr | II | ||
Cryptococcal meningitis | ≥13 yr | II | ||
Chronic granulomatous disease | Any age | IV | ||
|
||||
34. | Laronidase (Aldurazyme) (125058) 04/30/2003 |
Mucopolysaccharidosis I, multiple studies | All ages | I, II, III, IV |
|
||||
35. | Natalizumab (Tysabri) (125104) 11/23/2004 |
Crohn’s disease | 12 to 17 yr | II |
|
||||
36. | Ofatumumab (Arzerra) (125326) 10/26/2009 |
Leukemia | All ages | II |
|
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
37. | Omalizumab (Xolair) (103976) 06/20/2003 |
Asthma, multiple trials for moderate or severe asthma | ≥ 6 yr or ≥12 yr | III, IV |
Milk allergy | 4 to 18 yr | n/s | ||
Eosinophilic esophagitis | 12 to 60 yr | n/s | ||
12 to 76 yr | I | |||
Lung disease | ≥12 yr | IV | ||
Urticaria | 12 to 75 yr | II | ||
12 to 75 yr | III | |||
12 to 75 yr | III | |||
12 to 75 yr | III | |||
Cystic fibrosis | ≥12 yr | IV | ||
Hyper-immunoglobulin E syndrome | 6 to 76 yr | I | ||
Gastroenteritis | 12 to 76 yr | II | ||
Peanut allergy | 6 to 75 yr | II | ||
6 to 75 yr | II | |||
≥12 yr | I/II | |||
Atopic dermatitis | 12 to 60 yr | IV | ||
|
||||
38. | Oprelvekin (Neumega) (103694) 11/25/1997 |
Stem cell transplantation in malignancies | All ages | II |
Solid tumors | Up to 45 yr | I | ||
|
||||
39. | Palifermin (Kepivance) (125103) 12/15/2004 |
Mucositis | 1 to 16 yr | II |
Mucositis | 2 to 18 yr | I | ||
Leukemia | 1 to 16 yr | I | ||
Severe combined immunodeficiency | 2 to 20 yr | I/II | ||
Acute myeloid leukemia; advanced myelodysplastic syndromes | Up to 65 yr | II | ||
Lymphoma | 12 to 65 yr | I | ||
Graft-versus-host disease | 3 to 65 yr | I/II | ||
Mucositis | 12 to 65 yr | II | ||
Graft-versus-host disease | 3 to 65 yr | I/II | ||
Lymphoma | 12 to 70 yr | II | ||
Epidermolysis bullosa | Up to 21 yr | 0 | ||
Severe combined immunodeficiency | 18 mo to 20 yr | I | ||
|
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
40. | Palivizumab (Synagis) (103770) 06/19/1998 |
Respiratory syncytial virus infection | Up to 2 yr | |
Unhealthy children with a history of prematurity | 5 to 6 mo | II | ||
Airway hyperreactivity | 3 to 6 yr | |||
Chronic lung disease | Up to 24 mo | IV | ||
Healthy, previously dosed children | Up to 24 mo | I/II | ||
Heart disease | Up to 24 mo | III | ||
Pain from palivizumab injection | 1 mo to 2 yr | IV | ||
Recurrent wheezing | 3 mo to 1 yr | n/s | ||
|
||||
41. | Panitumumab (Vectibix) (125147) 09/27/2006 |
Solid tumors | 1 to 17 yr | I |
|
||||
42. | Pegfilgrastim (Neulasta) (125031) 01/31/2002 |
Solid malignancies | Up to 18 yr | II |
Solid malignancies | Up to 18 yr | II | ||
Type 1 diabetes | 12 to 45 yr | I/II | ||
Sarcoma | Up to 21 yr | II | ||
|
||||
43. | Peginterferon alfa-2A (Pegasys) (103964) 10/16/2002 |
Hepatitis B | 3 to 17 yr | III |
Hepatitis C | 5 to 18 yr | III | ||
Hepatitis C; hemophilia | ≥12 yr | IV | ||
Hepatitis C; thalassemia | ≥12 yr | IV | ||
Polycythemia vera or essential thrombothycemia | ≥18 wk | II | ||
Hepatitis C | 15 to 65 yr | III | ||
|
||||
44. | Peginterferon alfa-2A; ribavirin (Pegasys Copegus combination) (125083) 06/04/2004 |
Hepatitis C Hepatitis C |
5 to 18 yr >12 yr |
III IV |
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
45. | Peginterferon alfa-2B (Pegintron) (103949) 01/19/2001 |
Neurofibromatosis | 18 mo to 21 yr | II |
Malignant melanoma | Up to 21 yr | II | ||
Sarcoma | 5 to 40 yr | III | ||
HIV infection | 3 mo to 16 yr | I | ||
Plexiform neurofibroma | 1 to 21 yr | I | ||
Plexiform neurofibroma | 18 mo to 21 yr | II | ||
Neurofibromatosis | 2 to 30 yr | II | ||
Chronic myeloid leukemia | ≥12 yr | I | ||
Hepatitis C | 3 to 24 yr | III | ||
HIV infection | ≥15 yr | II | ||
Glioma | Up to 21 yr | II | ||
46. | Pegloticase (Krystexxa) (125293) 09/14/2010 |
|||
47. | Ranibizumab (Lucentis) (125156) 06/30/2006 |
|||
48. | Rasburicase (Elitek) (103946) 07/12/2002 |
Hyperuricemia | Up to 18 yr | IV |
Leukemia; lymphoma | 1 to 29 yr | II | ||
Malignancy-induced hyperuricemia | Age not specified | IV | ||
Tumor lysis syndrome | Up to 18 yr | IV | ||
Tumor lysis syndrome | ≥2 yr | n/s | ||
Nutritional and metabolic diseases | Up to 18 yr | II | ||
Leukemia; lymphoma | ≥15 yr | III | ||
Hyperuricemia | 1 to 75 yr | III | ||
Mature B-cell lymphoma | Up to 20 yr | II/III | ||
49. | Rilonacept (Arcalyst) (125249) 02/27/2008 |
Juvenile idiopathic arthritis | 18 mo to 19 yr | II |
Familial Mediterranean fever | ≥4 yr | II | ||
Cryopyrin-associated periodic syndromes | ≥7 yr | III | ||
50. | RimabotulinumtoxinB (Myobloc) (103846) 12/08/2000 |
Cerebral palsy (hand functioning) | 2 to 18 yr | I/II |
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
51. | Rituximab (Rituxan) (103705) 11/26/1997 |
Leukemia; lymphoma Lymphoproliferative disorder |
Six trials specifically include young patients; others include patients of any age | |
Neuroblastoma | 6 mo to 21 yr | n/s | ||
2 mo to 18 yr | ||||
Hemophilia | ≥18 mo | II | ||
Thrombotic thrombocytopenic purpura | ≥12 yr | III | ||
>12 yr | II/II | |||
≥12 yr | II | |||
Focal segmental glomerulosclerosis | 5 to 60 yr | II | ||
2 to 80 yr | ||||
Transplant-related complications, multiple trials | Various age ranges across pediatric population | II, III, IV | ||
Type 1 diabetes mellitus | 8 to 45 yr | II/III | ||
8 to 45 yr | IV | |||
Myositis | ≥5 yr | II | ||
Immunoglobulin A nephropathy | ≥5 yr | IV | ||
Nephrotic syndrome | 2 to 18 yr | II/III | ||
Wegener’s granulomatosis | ≥15 yr | II/III | ||
Aplastic anemia | ≥12 mo | n/s | ||
≥2 yr | II | |||
Neuromyelitis optica | 12 to 86 yr | I | ||
Central nervous system tumor | 18 mo to 75 yr | II | ||
Opsoclonus-myoclonus syndrome | 6 mo to 19 yr | I/II | ||
Chronic focal encephalitis | 5 to 25 yr | I | ||
Systemic lupus erythematosus | 15 to 40 yr | II | ||
Lymphomatoid granulomatosis | ≥12 yr | n/s | ||
|
||||
52. | Romiplostim (Nplate) (125268) 08/22/2008 |
Idiopathic thrombocytopenic purpura | 12 mo to 17 yr | III |
Idiopathic thrombocytopenic purpura | 12 mo to 17 yr | I/II | ||
Idiopathic thrombocytopenic purpura | 1 to 18 yr | III | ||
Idiopathic thrombocytopenic purpura | ≥1 yr | III | ||
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
53. | Tenecteplase (Tnkase) (103909) 06/02/2000 |
Restoration of function in dysfunctional central venous catheters (2 studies, including subjects weighing <10 kg) | n/s | III |
54. | Tocilizumab (Actemra) (125276) 01/08/2010 |
Systemic juvenile idiopathic arthritis | Up to 19 yr | III |
Systemic juvenile idiopathic arthritis | 2 to 17 yr | III | ||
Systemic juvenile idiopathic arthritis | Up to 24 mo | I | ||
55. | Tositumomab; iodine I 131 tositumomab (Bexxar) (125011) 06/27/2003 |
|||
56. | Trastuzumab (Herceptin) (103792) 10/25/1998 |
Osteosarcoma Recurrent osteosarcoma |
<30 yr Any age |
II II |
57. | Ustekinumab (Stelara) (125261) 09/25/2009 |
Psoriasis | 12 to 18 yr | III |
Biologics Under the Jurisdiction of the Center for Biologics Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
1. | Albumin (human) (Albumin) (125154) 10/17/2006 |
Cardiac surgery | 2 to 12 yr | IV |
Cardiac surgery | Up to 36 mo | n/s | ||
2. | Alpha1-proteinase inhibitor (human) (Aralast NP) (125039) 05/04/2007 |
Type 1 diabetes mellitus | 8 to 35 yr | II |
Type 1 diabetes mellitus | 8 to 35 yr | II | ||
Type 1 diabetes mellitus | 6 to 45 yr | I | ||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
3. | Alpha1-proteinase inhibitor (human) (Glassia) (125325) 07/01/2010 |
Type 1 diabetes mellitus | 10 to 25 yr | I/II |
4. | Alpha1-proteinase inhibitor (human) (Zemaira) (125078) 07/08/2003 |
|||
5. | Antihemophilic factor (recombinant), plasma/albumin free method (Advate) (125063) 07/25/2003 |
Hemophilia A (multiple studies) | Age ranges vary for specific studies but collectively cover the pediatric age range | I, II, III, IV |
6. | Antihemophilic factor (recombinant) (ReFacto) (103779) 03/06/2000 |
|||
7. | Antihemophilic factor (recombinant), plasma/albumin free (Xyntha) (125264) 02/21/2008 |
|||
8. | Antithrombin (recombinant) (ATryn) (125284) 02/06/2009 |
Postoperative hemorrhage in cardiopulmonary bypass surgery | Up to 30 days | I |
Postoperative hemorrhage in cardiopulmonary bypass surgery | Up to 30 days | III | ||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
9. | Anti-thymocyte globulin (rabbit) (thymoglobulin) (103869) 12/30/1998 |
Transplant-related complications, multiple trials | Various age ranges across pediatric population | I, II, III, IV |
Type 1 diabetes mellitus | 12 to 45 yr | I, II | ||
12 to 35 yr | II | |||
Aplastic anemia | ≥2 yr | II | ||
≥12 yr | II | |||
≥15 yr | II | |||
Systemic sclerosis | Up to 64 yr | II | ||
Myelodysplastic syndrome | All ages | II | ||
Toxicities of total body irradiation | Up to 21 yr | IV | ||
10. | Autologous cultured chondrocytes (Carticel) (103661) 08/22/1997 |
|||
11. | Botulism immune globulin intravenous (human) (BabyBIG) (125034) 10/23/2003 |
Infant botulism | Up to 1 yr | n/s |
12. | C1 esterase inhibitor (human) (Berinert) (125287) 10/09/2009 |
Hereditary angioedema | ≥6 yr | II/III |
Hereditary angioedema | ≥6 yr | III | ||
13. | C1 esterase inhibitor (Cinryze) (125267) 10/10/2008 |
Hereditary angioedema Hereditary angioedema |
2 to 11 yr ≥6 yr |
II II |
14. | Coagulation factor VIIa (recombinant) (NovoSeven) (103665) 03/25/1999 |
Hemophilia A | Up to 8 yr | II |
Cardiopulmonary bypass | Up to 30 days | n/s | ||
Hemophilia A | ≥2 yr | n/s | ||
Hemophilia | Up to 20 yr | IV | ||
Hemophilia A, B | ≥2 yr | II | ||
Hemophilia A, B | ≥2 yr | n/s | ||
Factor VII deficiency | Up to 90 yr | n/s | ||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
15. | Coagulation factor IX (recombinant) (Benefix) (103677) 02/01/1997 |
Hemophilia B (multiple studies) | Age ranges vary for specific studies but collectively cover the pediatric age range | III, IV |
16. | Crotalidae polyvalent immune Fab (ovine) (CroFab) (103788) 10/02/2000 |
Snakebite | 2 to 80 yr | III |
Snakebite | ≥1 yr | IV | ||
17. | Digoxin immune Fab (ovine) (DigiFab) (103910) 08/31/2001 |
|||
18. | Fibrin sealant (human) (Artiss) (125266) 03/21/2008 |
|||
19. | Fibrin sealant (human) (Evicel) (125010) 03/21/2003 |
Surgical blood loss | n/s | III |
20. | Fibrin sealant (TachoSil) (125351) (04/02/2010) |
Local bleeding, liver surgery | Up to 6 yr | II/III |
Local bleeding, liver surgery | All ages | III | ||
21. | Fibrin sealant (Tisseel) Baxter (103980) 05/01/1998 |
Burns | ≥6 yr | I/II |
Burns | Up to 65 yr | III | ||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
22. | Fibrinogen concentrate (human) (RiaSTAP) (125317) 01/16/2009 |
Cardiac surgical procedures | Up to 18 yr | II |
Fibrinogen deficiency | ≥6 yr | II | ||
23. | Hepatitis B immune globulin intravenous (human) (HepaGam B) (125237) 04/06/2007 |
|||
24. | Hepatitis B immune globulin (human) (Nabi-HB) (103945) 10/23/2001 |
|||
25. | Immune globulin intravenous (human) (Flebogamma 5% DIF [dual inactivation plus nanofiltration]) (125077) 12/15/2003 |
Trials for infections (both bacterial and viral); pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections; neonatal infection; recurrent infections and immunoglobulin G subclass deficiency; HIV infection; Rasmussen encephalitis | Various age ranges across the pediatric age spectrum | I, II, III, IV |
26. | Immune globulin intravenous (human) 10% solution (Gammagard liquid) (125105) 04/27/2005 |
Multiple trials for primary immunodeficiencies Trials for transplantation-related complications |
||
27. | Immune globulin intravenous (human) 5% liquid (Gammaplex) (125329) 09/17/2009 |
Other trials for abnormal muscle movement in neuroblastoma; sickle cell pain crisis; hyperbilirubinemia; idiopathic thrombocytopenic purpura; postpolio syndrome | ||
|
||||
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
|
||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
|
||||
28. | Immune globulin injection (human) 10% caprylate/chromatography purified (Gamunex-C) (125046) 08/27/2003 |
|||
|
||||
29. | Immune globulin subcutaneous (human) (IGSC) 20% liquid (Hizentra) (125350) 03/04/2010 |
|||
|
||||
30. | Immune globulin intravenous (human) 5% liquid (Octagam) (125062) 05/21/2004 |
|||
|
||||
31. | Immune globulin intravenous (human) 10% liquid (Privigen) (125201) 07/26/2007 |
|||
|
||||
32. | Immune globulin subcutaneous (human) (Vivaglobin) (125115) 01/09/2006 |
|||
|
||||
33. | Protein C concentrate (human) (Ceprotin) (125234) 03/30/2007 |
Protein C deficiency | ≤6 mo | II/III |
Protein C deficiency | n/s | IV | ||
|
Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
34. | Rho(D) immune globulin intravenous (human) (Rhophylac) (125070) 02/12/2004 |
|||
35. | Sipuleucel T (Provenge) (125197) 04/29/2010 |
|||
36. | Thrombin, topical (human) (Evithrom, a component of Evicel) (125247) 08/27/2007 |
Aid to hemostasis during surgery Aid to hemostasis during skin graft surgery |
Up to 17 yr 2 to 75 yr |
IV II |
37. | Thrombin, topical (recombinant) (Recothrom) (125248) 01/17/2008 |
|||
38. | Vaccinia immune globulin intravenous (CNJ-016) (125109) 05/02/2005 |
Corneal scarring associated with vaccinia complication | ≥1 yr | II |
Prevention of vaccinal infection | n/s | I | ||
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Biologics Under the Jurisdiction of the Center for Drug Evaluation and Research | ||||
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Generic Name (Trade Name) (BLA Number) Original Approval Date |
Condition | Ages of Trial Participants | Trial Phase | |
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39. | von Willebrand factor/coagulation factor VIII complex (human) (Wilate) (125251) 12/04/2009 |
Bleeding prevention in surgery | ≥6 yr | III |
Von Willebrand disease | n/s | n/s | ||
Hemophilia A | Any age | n/s | ||
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a For the hepatitis B immune globulin products, none of the pediatric study listings involving this type of product cited either brand name.
NOTES: For age, n/s indicates a study for which the trial description did not state age explicitly but included children’s hospital sites or had inclusion criteria or other information text that indicated the inclusion of pediatric patients (e.g., references to trial patients <10 kg). For trial phase, n/s indicates that the phase was not specified in the description. Search terms included a combination of the generic “biologic name AND children” or the “trade name AND children” to capture all registered studies that used that agent. Some biologic agents that are often treated as interchangeable have been grouped together by their generic name (e.g., immune globulin intravenous). For each product that has relevant studies for the class of drug, at least one study identifies that brand name. The listings for each product may not be exhaustive of trials for the same condition, age group, and phase.
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