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Orphan Exclusivity Dilemma: Without Legislative Fix, US FDA May Seek New Regulation

 
• By Brenda Sandburg

FDA didn’t get policy rider it wanted which would have reversed the Catalyst court ruling that the agency cannot limit a drug’s orphan exclusivity to its approved indication. One attorney says the clearest path forward is for FDA to issue new rulemaking and face additional litigation.

orphan FDORA
FDA may propose new regulation on orphan drug exclusivity • Source: Shutterstock

The US Food and Drug Administration had hoped to get around an appeals court ruling that rejected its interpretation of the Orphan Drug Act, but after an effort to win a legislative reversal failed, the agency now faces the dilemma of how to award orphan exclusivities under the new scheme as well as address the backlog of determinations that have been pending since the unfavorable Catalyst court decision. 

FDA seemed like it was halfway home after the House version of the user fee reauthorization bill emerged in the spring with a provision backing FDA’s position that orphan drug exclusivity only applies to the specific indication or use for which the drug is approved, not the entire disease

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