More than a year has passed since the US Court of Appeals for the Eleventh Circuit's decision in Catalyst Pharms., Inc. v. Becerra struck down the US Food and Drug Administration's (FDA) indication-specific interpretation of orphan drug exclusivity (ODE).1 In response to that September 2021 decision, FDA's Office of Orphan Products Development (OOPD) has deferred all of its pending orphan exclusivity determinations. In fact, OOPD has not publicly recognized orphan exclusivity for any product approved since November 2021. Rather than recognize a broad, disease-specific scope of orphan exclusivity consistent with the Eleventh Circuit's opinion, the agency appears to be waiting for a legislative fix to effectively overrule the Catalyst decision. The status of that legislative fix is in jeopardy, however, in light of the recent passage2 of a "clean" version of the FDA user fee reauthorization provisions'without any changes to the Orphan Drug Act. As a result, sponsors remain in the dark about the impact of the court decision, and FDA has given little indication how the agency intends to proceed.
The Catalyst Decision
Briefly, the Catalyst decision held that FDA's indication-specific interpretation of the scope of ODE was incompatible with the Orphan Drug Act's exclusivity provision, which describes a "rare disease or condition." Under FDA's historical approach, which had been codified in agency regulations since 2013, a drug product approved for an "indication or use" that is narrower than the "rare disease or condition" for which the orphan designation was granted would obtain exclusivity only for the approved indication or use. However, the Eleventh Circuit held in Catalyst that the scope of exclusivity was as broad as the rare disease or condition for which designation was granted, even if a drug product's approval was for a narrower indication. In the context of the Catalyst case, the court thus concluded that the orphan exclusivity for Catalyst's Firdapse (amifampridine phosphate) blocked approval of Jacobus Pharmaceutical Co.'s (Jacobus's) competitor product, Ruzurgi (amifampridine), for a different indication.3
The Aftermath, Part 1: Catalyst and Jacobus Settle Their Dispute, Leaving Firdapse the Sole Product on the Market
Following the Eleventh Circuit decision, intervenor-defendant Jacobus initially submitted a petition for a writ of certiorari to the Supreme Court in hopes of overturning the appellate court's decision. FDA did not join or otherwise...
