Catalyst Pharmaceuticals, Inc. has recently announced that its sub-licensee in Japan, Dydo Pharma, Inc., has received approval from the Ministry of Health, Labor and Welfare of Japan to commercialize Firdapse® (amifampridine) tablets 10 mg for the treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS) in Japan.
This approval signifies a significant milestone in addressing the unmet needs of patients affected by LEMS in Japan, as it brings renewed hope to patients and affirms Firdapse's proven effectiveness in treating LEMS. Firdapse is already an approved treatment for LEMS in the United States, where it is indicated for adults and pediatric patients six years of age and older.
LEMS is a rare autoimmune disorder characterized by muscle weakness and fatigue, and about 50% of people with LEMS have underlying cancer, as it is observed in approximately 3% of small-cell lung cancer patients. Firdapse is the only U.S. FDA-approved, evidence-based treatment for LEMS and is supported by a comprehensive patient support program to ensure accessibility and assistance for eligible U.S. patients.
Firdapse's mechanism of action involves causing depolarization of the presynaptic membrane and slowing or inhibiting repolarization, which results in the opening of slow voltage-dependent calcium channels, allowing for an influx of calcium. This action induces the release of acetylcholine into the synaptic cleft, enhancing neuromuscular transmission and improving muscle function.
Dydo Pharma, the rare disease pharmaceutical subsidiary of Dydo Group Holdings, will be responsible for commercializing Firdapse in Japan. Dydo Group Holdings, Inc. operates through various segments, with the domestic beverage business accounting for more than 70% of total sales.
Catalyst Pharmaceuticals, Inc., a biopharmaceutical company committed to improving the lives of patients with rare diseases, has a well-established presence in the U.S. and is actively seeking to expand its global commercial footprint through strategic partnerships.
The approval of Firdapse in Japan represents a significant step forward for Catalyst Pharmaceuticals, Inc., as it continues to prioritize accessibility and patient care in its efforts to bring life-changing treatments to the market. Today the company's shares have moved 0.0% to a price of $20.0. For more information, read the company's full 8-K submission here.
