Clarification of Orphan-Drug Exclusivity Following Catalyst Pharms., Inc. v. Becerra; Notification

The Food and Drug Administration (FDA or Agency) is publishing this notification in light of the recent decision by the U.S. Court of Appeals for the Eleventh Circuit in Catalyst Pharms., Inc. v. Becerra. The Catalyst decision addressed the orphan-drug exclusivity provision of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by the Orphan Drug Act and subsequent amendments, and concluded that FDA's approval of Jacobus Pharmaceutical Company's (Jacobus's) drug (the drug at issue in the litigation) must be set aside. Consistent with the court's decision, FDA has set aside its approval of Jacobus's drug. This notification announces that, at this time, while complying with the court's order in Catalyst, FDA intends to continue to apply its regulations tying the scope of orphan-drug exclusivity to the uses or indications for which a drug is approved to matters beyond the scope of that order.