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# Courts Forcing FDA to Reconsider Orphan Drug Designation: Analysis
- URL: https://www.fdaweb.com/courts-forcing-fda-to-reconsider-orphan-drug-designation-analysis/
- Published: 2021-10-05T12:00:00.000Z
- Updated: 2026-09-14T17:17:39.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5150292

Recent appeals court decisions against FDA in orphan drug cases will “force FDA to take a hard look at this approach to orphan drug designations, as it may find it difficult to develop a consistent rubric for evaluating orphan drug designation requests.” That’s the view of attorneys **Sara Koblitz** and **Kurt Karst** (Hyman, Phelps & McNamara), [commenting](https://www.thefdalawblog.com/2021/10/oops-fda-did-it-again-another-orphan-drug-act-loss-for-fda-based-on-unambiguous-statutory-text-11th-circuit-rules-that-the-scope-of-orphan-drug-exclusivity-is-determined-by-the-rare-disease-or/?ref=fdaweb.com) in their firm’s *FDA Law Blog* on an 11th Circuit Court of Appeals decision that the agency’s interpretation of the Federal Food, Drug, and Cosmetic Act is contrary to the plain text of the statute.

In *Catalyst v. FDA*, the appeals court reversed a Florida federal court [decision](https://www.fdaweb.com/appeals-court-reverses-catalyst-firdapse-loss-to-fda/) that the statutory phrase “same disease or condition” in the Orphan Drug Act is ambiguous. The appeals court said the phrase was not ambiguous merely because Congress did not provide an explicit definition in the law.

Koblitz and Karst say the ruling may lead to new litigation or call into question previous agency awards of orphan drug exclusivity. “In some cases,” they write, “particularly where drugs were designated and approved prior to this case, orphan drug exclusivity may now extend significantly farther than the approved indication for a product and the holder of another marketing application for the same drug for a different indication that reads on the same rare disease or condition in jeopardy of a challenge or FDA having to yank the approval. In other cases, however, there may be challenges to FDA’s award of multiple periods of orphan drug exclusivity for the same drug for different indications of the same rare disease or condition.”

The attorneys say it will be difficult for the agency to retool the orphan drug designation process to ensure that orphan drug development is properly incentivized while not providing a windfall to sponsors whose approved drug is not as helpful for a designated condition as hypothesized.