Catalyst Pharmaceuticals, Inc. is leveraging the expanded access protocol for amifampridine phosphate to provide treatment for pediatric patients with Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS). The approval for marketing of Firdapse® for adult patients with LEMS in November 2018 and for pediatric patients aged 6 years and older in September 2022 positions the company favorably within the niche market of rare neuromuscular disorders. The ongoing expanded access study allows for the collection of long-term safety data, which may enhance the product's value proposition and support future regulatory submissions for additional indications. The competitive landscape includes other neuromuscular agents, but amifampridine's unique mechanism and established safety profile may provide a competitive edge.