(2006), acute lymphoblastic leukemia (2006), and dermatofibrosarcoma (2006). Imatinib is therefore a single orphan drug with seven different disease-based approvals. To cite a different situation, the combination product benzoate-phenylacetate was initially approved in 1987 as Ucephan (Immunex Corp.), an oral formulation for management of hyperammonemia, but the manufacturer later withdrew it from the market. Another company then sought new approval as an orphan product for the same indication (but in an intravenous formulation) under the name Ammonul (Ucyclyd, a subsidiary of Medicis Pharmaceutical Corp.) in 2005. For the purposes of analyzing the impact of the Orphan Drug Act in this study, benzoate-phenylacetate counted as two separate products because it appears to have originated from two separate manufacturers and also to differ in formulation.

This process identified a subset of 279 separate orphan products among the original sample that were approved for 347 designations or indications. Within this subset, 233 products had a single approved orphan designation, 36 products had two designations, 5 products had three designations, 3 drugs had four designations, and 2 products had seven designations each

TABLE B-1 Orphan Approvals for Somatropin Products (human growth hormone, hGH)

Brand Name

Year Approved

Manufacturer(s)

Comments

Separate Product?

Nutropin

1985

Genentech

 

Y

Protropin

1985

Genentech

Identical to Nutropin, except for single amino acid on the N-terminus of the molecule

N

Humatrope

1987

Lilly

 

Y

Serostim

1996

Serono

 

Y

Saizen

1996

Serono

Designated as an orphan but not granted market exclusivity. Structurally equivalent to Serostim, but given a different brand name for a different indication

N

Genotropin

1997

Pharmacia and Upjohn

 

Y

Nutropin Depot

1999

Genentech

New delivery system and slightly different formulation

Y

Zorbtive

2003

Serono

Same structure as Serostim and same manufacturer, although given different brand name for different orphan indication

N

Norditropin

2007

Novo Nordisk

Originally approved in 1995

Y



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