Catalyst Pharmaceuticals Inc is a commercial stage patient centric biopharmaceutical company focused on in licensing developing and commercializing novel medicines for patients living with rare and difficult to treat diseases. The company sells three commercial stage drug products FIRDAPSE amifampridine AGAMREE vamorolone and FYCOMPA perampanel. It seeks to expand its product portfolio by acquiring rights to immediate and near term accretive assets that treat orphan diseases across therapeutic areas including clinical stage opportunities with established proof of concept. Catalyst maintains a patient centric approach in all its activities and supports its commercial efforts with a field force of approximately 58 individuals dedicated to FIRDAPSE and AGAMREE plus 19 patient assistance liaisons and 19 medical science liaisons. After launching AGAMREE in March 2024 the company separated its field based forces into two distinct units one for each product effective April 1 2025. It also employs two national account directors who concentrate on the oncology market for LEMS patients who also have cancer.

Catalyst generates revenue primarily from the sale of its three approved products in the United States. FIRDAPSE is sold domestically through a specialized field force and its distribution is supported by the Catalyst Pathways patient support program and a network of exclusive specialty pharmacies chiefly AnovoRx. The company also earns revenue from sublicensees in Canada and Japan where KYE Pharmaceuticals and DyDo Pharma market FIRDAPSE under agreements that include milestone payments and royalties based on net sales. AGAMREE is sold in the United States through a dedicated field team and the same Catalyst Pathways program while the company holds an exclusive North American license from Santhera and owes milestone and royalty payments tied to AGAMREE sales and to Santhera for development and commercialization milestones. In Canada AGAMREE is marketed by KYE under a similar sublicense arrangement that provides Catalyst with sales milestones and royalties. FYCOMPA was acquired from Eisai and its revenue comes from product sales despite the loss of patent exclusivity although the company ceased active marketing efforts for FYCOMPA at the end of 2025. Eisai continues to manufacture FYCOMPA under a long term supply agreement and Catalyst pays Eisai for the finished product. Additionally Catalyst has received upfront payments and milestone payments related to the licensing and investment agreements with Santhera for AGAMREE and with Eisai for FYCOMPA.

Catalyst operates in the highly competitive orphan drug market where it faces rivals that offer alternative therapies for the same rare indications. In the LEMS space the principal competitors include Jacobus Pharmaceuticals with its product Ruzurgi and off label use of pyridostigmine as well as immunosuppressive agents such as steroids azathioprine and intravenous immunoglobulin. For DMD the company competes against established corticosteroid treatments like prednisone and deflazacort marketed as Emflaza as well as newer non steroid options such as Givinostat and gene therapy Elevidys. In epilepsy FYCOMPA previously competed with a broad array of antiepileptic drugs but after the loss of exclusivity the company has stopped promoting the product and relies on existing sales. Catalysts competitive advantages stem from its patient centric support programs such as Catalyst Pathways which provides personalized dosing assistance and access to specialty pharmacies its strong intellectual property portfolio that includes multiple patents with extended expiration dates and its orphan drug exclusivity that protects FIRDAPSE until late 2025 and AGAMREE until 2030. The company also benefits from a solid cash position that enables it to pursue business development opportunities and to fund patient assistance initiatives.

Catalyst serves patients diagnosed with LEMS DMD and epilepsy as well as the physicians who treat these conditions. Its customer base includes neurologists who manage LEMS oncologists who treat small cell lung cancer and may encounter LEMS as a comorbidity and pediatric and adult neurologists who care for DMD patients. The company distributes its products through specialty pharmacies such as AnovoRx and works with patient assistance foundations and charitable organizations to help uninsured or underinsured patients obtain therapy. Catalyst also collaborates with rare disease advocacy groups including the Myasthenia Gravis Foundation of America the National Organization for Rare Disorders and the LEMS Family Association to raise disease awareness and educate healthcare providers. In addition the company maintains commercial relationships with its sublicensees KYE Pharmaceuticals in Canada and DyDo Pharma in Japan for FIRDAPSE and with Santhera for AGAMREE development and with Eisai for the manufacture of FYCOMPA.