FDA Pushes Congress for Catalyst Decision Fix
FDA is interested in working with Congress to find a legislative solution, most likely a bill that would be added to the user fee reauthorization package that is moving through Congress, to help overturn a 2021 appeals court decision that has disrupted FDA’s orphan drug program. Asked to comment on the decision in Catalyst Pharmaceuticals v. Becerra during a 4/26 hearing on reauthorizing user fees, CBER director Peter Marks said the decision has harmful implications for developing drugs in pediatric indications for rare diseases.
A Hogan Lovells legal analysis earlier this year said that the 11th Circuit Court of Appeals decision upholding orphan exclusivity for Catalyst Pharmaceuticals’ Firdapse (amifampridine phosphate) “appears to undermine FDA’s indication-specific approach to orphan exclusivity, providing exclusivity holders (and those on deck for approval) potentially broader exclusivity than previously expected.” In an online post, the attorneys also said the decision may significantly increase the burden for second-in-time sponsors seeking to avoid a competitor’s orphan exclusivity.
By regulation, the attorneys wrote, FDA has limited the scope of orphan exclusivity to only the approved indication or use, even if the underlying designation is for a broader disease or condition. “The impact of this decision may be straightforward with respect to the Jacobus product,” the post concludes, “but the implications for other sponsors are less clear. FDA faces a difficult decision in deciding whether and how to implement this decision with regard to other sponsors holding orphan designation or exclusivity.”
During the hearing, Senators Tammy Baldwin (D-WI) and Bill Cassidy( R-LA) both hinted that they are working on an amendment to the user fee legislation to curb orphan drug loopholes and are intending to address the predicament the Catalyst decision has created.
Baldwin asked CDER director Patrizia Cavazzoni to comment on the Catalyst decision too. “We share a concern and want to work with Congress to find solutions,” Cavazzoni replied. “The Catalyst decision will send a chill through the development of rare diseases and it will disproportionately affect children with rare diseases. It is essential that we continue to generate and spur the study of drugs in children, and so this decision will really go counter to that. The situation following that decision is that a sponsor could study a disease in a very narrow segment of the population and then be able to block further approvals throughout the entire condition that the drug could address. So, it is very concerning, and we appreciate Congress’ interest and look forward to working with you.”