Genentech, Inc. v. Bowen
1987 U.S. Dist. LEXIS 12624, 676 F. Supp. 301, 1987 WL 33570 (1987)
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Rule of Law:
The Orphan Drug Act permits the FDA to designate a synthetic drug as an 'orphan drug' for marketing exclusivity, even if chemically similar to a previously approved drug for the same condition, when the prior drug is no longer practically available due to safety concerns and the synthetic drug offers a critical safety advantage, thereby fulfilling the Act's purpose of providing treatments for rare diseases.
Facts:
- Human growth hormone (hGH) deficiency affects between 6,000 and 15,000 children in the United States.
- Since 1958, this condition had been treated by supplementing a patient’s natural hGH with hGH derived from the pituitary glands of human cadavers (pituitary-derived hGH).
- In 1985, the use of pituitary-derived hGH was effectively eliminated after three patients developed Creutzfeldt-Jakob Disease, apparently due to a pathogen transmitted by the pituitary-derived hGH.
- On October 17, 1985, the FDA granted Genentech marketing approval for Protropin, a synthetic human growth hormone (r-hGH) produced through recombinant DNA technology, which includes an additional methionine amino acid group.
- On December 12, 1985, the FDA designated Protropin as an orphan drug, granting Genentech marketing exclusivity until December 12, 1992.
- On June 12, 1986, the FDA designated Lilly’s r-hGH drug, Humatrope, as an orphan drug; Humatrope's chemical structure is identical to natural, pituitary-derived hGH, lacking the additional methionyl group found in Protropin.
- On October 15, 1986, Lilly submitted a New Drug Application (NDA) to the FDA for its Humatrope product, seeking permission to market the drug commercially.
- On March 8, 1987, the FDA approved Lilly’s NDA for Humatrope, thereby authorizing Lilly to market the drug commercially and triggering the orphan drug exclusivity provision.
Procedural Posture:
- On November 3, 1986, Genentech submitted a 'citizen petition' to the FDA, asserting that Lilly's drug was the 'same' as Protropin and ineligible for approval until 1992, and requesting new FDA procedures for determining drug sameness.
- Upon learning that the FDA was preparing to approve Lilly's NDA for Humatrope, Genentech sought an emergency stay from the FDA, which was denied.
- On March 6, 1987, Genentech filed suit in the U.S. District Court for the District of Columbia, seeking temporary, preliminary, and permanent injunctive relief, and a declaratory judgment that the FDA's application of the Orphan Drug Act violated its statutory and constitutional rights.
- The District Court denied Genentech's request for a temporary restraining order on March 6, 1987.
- On March 6, 1987, the FDA formally responded to Genentech’s citizen petition, denying the requests for implementation of new procedures and for a stay.
- Intervenor-defendant Ares-Serono, Inc. (Serono) and intervenor-plaintiffs Nordisk Gentoffe A/S and Nordisk-U.S.A. (Nordisk) joined the lawsuit.
- Genentech, Serono, and Nordisk filed separate motions for partial summary judgment, challenging the validity of the FDA's designation of Lilly's drug (Humatrope) as an orphan drug.
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Issue:
Does the Orphan Drug Act permit the FDA to designate a synthetic human growth hormone (r-hGH) as an 'orphan drug' for marketing exclusivity purposes, even though a pituitary-derived human growth hormone (p-hGH) had previously received marketing approval for the same condition, when the p-hGH is no longer available due to safety concerns and the r-hGH has distinct safety advantages?
Opinions:
Majority - Stanley S. Harris, District Judge
Yes, the Orphan Drug Act permits the FDA to designate a synthetic human growth hormone as an orphan drug under these circumstances. The court found that Humatrope and pituitary-derived hGH are not the same drug for the purposes of the Orphan Drug Act's designation provision (§ 360bb(a)). The legislative history of the Act reveals a broad, bipartisan goal of providing treatment for presently untreated patients. In this case, Humatrope, being synthetically derived, does not present the risk of Creutzfeldt-Jakob disease associated with cadaver-derived hGH. Furthermore, the withdrawal of pituitary-derived hGH from the U.S. market meant that, despite prior NDAs, no methionyl-free hGH was available. Congress clearly focused on the availability of treatments, not merely the technical existence of prior NDAs, when enacting the Orphan Drug Act. Therefore, in light of the critical safety advantage and the practical unavailability of the former treatment, the court concluded that Congress would have considered Humatrope sufficiently 'different' to justify orphan drug designation, upholding the FDA's decision.
Analysis:
This case highlights the judiciary's role in interpreting ambiguous statutory language ('drug' in this context) when an administrative agency has not promulgated clarifying regulations. The court relied heavily on legislative intent and the practical objectives of the Orphan Drug Act (encouraging the availability of treatments for rare diseases) to make its determination. This decision implies that safety profiles and the actual market availability of a drug can be crucial factors in determining whether a new version of a drug is 'different' enough to qualify for orphan drug benefits, even if chemically similar to a previously approved, but now unsafe or unavailable, product. It sets a precedent for a contextual, purpose-driven interpretation of 'drug' under the Orphan Drug Act, particularly when public health and market access are at stake.
