Catalyst Pharmaceuticals (NASDAQ:CPRX) is a biopharmaceutical company headquartered in Coral Gables, Florida, dedicated to developing and commercializing therapies for people living with rare neuromuscular, neurological and cholestatic liver diseases. Founded in 2002 under its former name, Neurobiologic Technologies, the company went public through an initial public offering in 2011 and has since focused its research and development efforts on addressing conditions with high unmet medical needs. The leadership team is spearheaded by Chief Executive Officer Stephen Farr and Chief Financial Officer Michael Andree, who bring extensive industry experience in rare disease drug development and commercialization.
The company’s flagship product is FIRDAPSE® (amifampridine) tablets, which received accelerated approval from the U.S. Food and Drug Administration for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder that affects neuromuscular transmission. Catalyst obtained full approval for FIRDAPSE in 2018 following successful completion of confirmatory clinical trials, and the therapy is now available by prescription throughout the United States. In addition to its commercial operations, Catalyst works closely with patient advocacy groups and specialty pharmacies to support access and education for LEMS patients and healthcare providers.
In pursuit of expanding its portfolio, Catalyst has also advanced a pipeline of investigational medicines targeting additional rare diseases. The most advanced candidate is maralixibat, an ileal bile acid transporter inhibitor licensed for development in cholestatic liver diseases such as Alagille syndrome and progressive familial intrahepatic cholestasis. These programs are supported by ongoing clinical trials and strategic collaborations aimed at leveraging Catalyst’s expertise in rare disease development. The company continues to evaluate in-licensing and acquisition opportunities to enhance its pipeline and address other underserved patient populations.
Operationally, Catalyst Pharmaceuticals manages research and regulatory activities from its headquarters in Florida, while partnering with contract development and manufacturing organizations to produce clinical and commercial supplies. The company’s business model emphasizes patient-centric development, close engagement with regulatory authorities and efficient commercialization strategies for rare disease products. With a growing portfolio and a clear focus on specialized therapies, Catalyst aims to deliver meaningful treatment options for patients facing challenging, life-altering conditions around the world.
