Catalyst Pharmaceuticals is a biopharmaceutical company based in Coral Gables, Florida. The company is developing therapeutics for rare neurological diseases, including Firdapse (3,4-diaminopyridine phosphate, a potassium channel blocker) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) and CPP-115 (a GABA transaminase) for the treatment of infantile spasms.

Founded in 2002, Catalyst Pharmaceuticals competed an IPO in 2006. Catalyst focused primarily on developing therapies to prevent addiction until 2012.

In 2009, Catalyst Pharmaceuticals in-licensed worldwide rights to a family of GABA inhibitors including CPP-115 from Northwestern University. Catalyst Pharmaceuticals in-licensed the right to develop Firdapse for the North American market from BioMarin in 2012.

Catalyst Pharmaceuticals obtained a breakthrough therapy designation for Firdapse from the FDA for the development of a treatment of patients suffering from LEMS in 2013. Catalyst Pharmaceuticals has evaluated Firdapse for safety and efficacy in clinical trials. Although not approved by the FDA as of 2015, Firdapse is available to eligible LEMS patients through an Expanded Access Program. Firdapse is also being evaluated as a potential therapy for patients with congenital myasthenic syndromes, for which it has obtained an orphan drug designation from the FDA.

Catalyst Pharmaceuticals is also developing CPP-115 as a treatment for infantile spasms.

The development of Firdapse by Catalyst Pharmaceuticals has brought attention to orphan drug policies that grant market exclusivity as an incentive for companies to develop therapies for small numbers of patients.