## Facts

- Genentech marketed Protropin, a recombinant human growth hormone (hGH), and held orphan-drug designation/approval for pediatric growth hormone deficiency indications.
- Earlier hGH therapies in the United States were pituitary-derived (cadaver-extracted) and had been approved under NDAs for similar rare growth disorders.
- Pituitary-derived hGH was withdrawn from clinical use due to contamination risk associated with Creutzfeldt-Jakob disease.
- Eli Lilly developed Humatrope, a recombinant hGH intended for the same rare pediatric growth hormone deficiency indications.
- Before marketing approval, FDA designated Humatrope as an orphan drug under the Orphan Drug Act (ODA).
- Genentech (joined by other manufacturers) sued federal health officials under the APA, the ODA, and the Fifth Amendment, seeking to invalidate the designation and to protect its asserted exclusivity.
- The court considered separate motions for partial summary judgment challenging the legality of FDA’s pre-approval orphan designation for Humatrope.

## Issues

1. Whether FDA could lawfully designate Humatrope as an orphan drug despite Genentech’s prior orphan designation/approval for recombinant hGH for the same rare indications.
2. Whether existing NDAs for pituitary-derived hGH (and Genentech’s orphan exclusivity) barred FDA from designating and prospectively approving Humatrope for the same indication.
3. Whether FDA’s designation decision was arbitrary or capricious under the APA or violated Genentech’s Fifth Amendment rights.

## Decision

- The court denied the motions for partial summary judgment.
- The court upheld FDA’s orphan-drug designation of Humatrope.
- The court accepted FDA’s position that pituitary-derived hGH and recombinant hGH were different drugs for ODA purposes, so earlier pituitary-derived NDAs did not bar designation.
- The court concluded FDA’s construction and application of the ODA were permissible and not arbitrary or capricious.
- The court rejected the Fifth Amendment challenges.

## Legal Principles

- Under APA review, FDA action implementing the ODA is upheld if the agency’s interpretation is reasonable and its application is not arbitrary, capricious, or contrary to law.
- The ODA permits FDA to treat products as different drugs based on medically and scientifically meaningful distinctions, including safety profile and method of manufacture.
- Prior approval history does not automatically preclude a later orphan designation when FDA reasonably determines the later product is a different drug for the same rare condition.
- The ODA does not condition orphan designation or exclusivity on the sponsor’s profitability or subjective reliance on statutory incentives.

## Conclusion

The court sustained FDA’s pre-approval orphan designation for Humatrope, holding that FDA reasonably treated recombinant hGH as different from withdrawn pituitary-derived hGH and permissibly applied the ODA and APA standards without violating Genentech’s constitutional claims.


Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987)