FDA to stay the course on orphan exclusivity post-Catalyst

Regulatory NewsRegulatory News | 24 January 2023 |  By 

The US Food and Drug Administration (FDA) will continue to apply orphan drug exclusivity according to its regulations, as it did prior to the Catalyst Pharms., Inc. v. Becerra ruling.
The agency’s “longstanding” interpretation of the Orphan Drug Act’s exclusivity provision was thrown into question following the Catalyst decision in September 2021.
Under the 1983 Orphan Drug Act, drug approved to treat rare diseases can get seven years of market exclusivity beginning from the time of new drug application (NDA) or biologics license application (BLA) approval. Orphan drug exclusivity blocks the agency from approving other drugs for the same orphan indication during the exclusivity period.
In 2019, Catalyst sued FDA over the agency’s decision to approve a drug from one of its competitors, Jacobus Pharmaceutical Company. Both companies held orphan drug designations for the same drug – amifampridine for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). FDA approved catalyst’s version of the drug in November 2018 to treat adults with the condition, and in May 2019, the agency approved Jacobus’ drug to treat LEMS in children.
FDA held that its regulations enable it to apply orphan drug exclusivity only to the approved use or indication within an eligible disease, and not to all uses or indications within entire disease or condition. While a district court sided with FDA and found that the agency “reasonably interpreted the statute,” Catalyst appealed the decision to the U.S. Court of Appeals for the Eleventh Circuit, which overturned the lower court’s decision and ordered FDA to set aside its approval of Jacobus’ drug.
Within a month of the ruling, FDA stopped issuing decisions about orphan drug exclusivity for drugs approved with the decision. According to the Federal Register notice, it will go forward in applying the policy as of Tuesday, 24 January 2023.
“At this time, while complying with the court’s order in Catalyst, FDA intends to continue to apply its regulations in 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,” FDA said.
FDA also insisted that its past interpretation of the statute was correct and carried out the intent of the Orphan Drug Act.
“FDA continues to believe that the statutory text does not unambiguously require that orphan-drug exclusivity extend to the entire disease or condition for which a drug received orphan-drug designation if the drug is only approved for some uses within that disease or condition. Further, FDA believes that its statutory interpretation embodied in its regulations best advances the Orphan Drug Act’s purposes, appropriately balancing the need to incentivize the development of drugs for rare diseases and conditions with the need to provide patient access to orphan drugs.”
FDA added that its regulations accomplish the goals of orphan drug exclusivity by incentivizing the development of drugs for new indications in the rare disease space.
“FDA believes that continued adherence to its validly promulgated regulations will best serve the public health by facilitating patient access to orphan drugs, especially for difficult-to-study patients such as young children,” the agency said.


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