11th Circuit Threatens FDA Orphan Drug Decisions: Attorneys
Four Hogan Lovells attorneys say an 11th Circuit Court of Appeals 1/28 mandate 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 say the decision may significantly increase the burden for second-in-time sponsors seeking to avoid a competitor’s orphan exclusivity.
By regulation, the attorneys write, FDA has limited the scope of orphan exclusivity only to the approved indication or use, even if the underlying designation is for a broader disease or condition.
In the 11th Circuit decision, Catalyst had obtained designation for Firdapse to treat Lambert-Eaton myasthenic syndrome (LEMS) in adults. At the time FDA approved Firdapse, it was reviewing an NDA from Jacobus Pharmaceutical Co. for Ruzurgi (amifampridine) for treating LEMS that sought an indication for both adult and pediatric patients.
FDA concluded that approval of Ruzurgi for treating LEMS in adults was blocked by the Firdapse orphan exclusivity. But it approved Ruzurgi for treating LEMS in pediatric patients 6 to less than 17 years old.
When a Florida federal court granted summary judgment in favor of FDA when Catalyst sued, the company appealed to the 11th Circuit, which ruled in its favor, saying the exclusivity provision unambiguously “prohibits the approval of subsequent NDAs for amifampridine to treat LEMS … while Catalyst holds its seven-year exclusivity.”
The appeals court remanded the case to the district court with instructions to grant judgment in favor of Catalyst. The attorneys say they expect the district court to either vacate the FDA approval of Ruzurgi or remand to the agency to implement the appeals court ruling.
“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.”