FDA Approves Higher Max Dose for Catalyst's FIRDAPSE®

Catalyst Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for FIRDAPSE® (amifampridine). This approval increases the indicated maximum daily dosage for both adults and pediatric patients weighing over 45 kg, from 80 mg to 100 mg. The drug is used to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder characterized by muscle weakness and fatigue. FIRDAPSE, a potassium channel blocker, enhances the release of acetylcholine at the neuromuscular junction, thereby improving muscle function in LEMS patients. It remains the only FDA-approved treatment for LEMS in the United States.

Richard J. Daly, President and CEO of Catalyst Pharmaceuticals, expressed satisfaction with the FDA's approval, noting that it offers more flexibility in treatment regimens for LEMS patients and aligns with the company's mission to optimize patient outcomes. Catalyst believes this milestone will significantly impact the lives of LEMS patients by providing new treatment options.

Patients in the U.S. can obtain FIRDAPSE through prescriptions from their healthcare providers. Catalyst also offers a Patient Assistance Program called Catalyst Pathways® for FIRDAPSE®, providing comprehensive support to eligible patients and their families throughout the treatment process.

Catalyst Pharmaceuticals focuses on developing and commercializing innovative medicines for rare and difficult-to-treat diseases. Its flagship product, FIRDAPSE® (amifampridine) 10 mg tablets, is approved for LEMS treatment in adults and children aged six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to FYCOMPA® (perampanel), a prescription medication for epilepsy. Health Canada has also approved FIRDAPSE for treating adult LEMS patients in Canada.

Furthermore, Catalyst has obtained an exclusive North American license for AGAMREE® (vamorolone) oral suspension, a novel corticosteroid for Duchenne Muscular Dystrophy. AGAMREE received FDA Orphan Drug and Fast Track designations and was approved for U.S. commercialization on October 26, 2023. It became available by prescription in the U.S. on March 13, 2024.

Catalyst Pharmaceuticals is committed to addressing the needs of patients with rare diseases through the development and commercialization of first-in-class medicines. The company aims to make meaningful advancements in patient care by providing innovative treatment solutions.