Catalyst Pharmaceuticals Announces Publication of Santhera Pharmaceutical’s VISION-DMD Vamorolone (AGAMREE) Study Results in the Peer-Reviewed Journal Neurology - Seite 3

About Duchenne muscular dystrophy (DMD)
DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 patients in the U.S. are affected by DMD, with approximately 70% of patients currently receiving concomitant corticosteroid treatment.

About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare and difficult to treat diseases. Catalyst's flagship U.S. commercial product is FIRDAPSE (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and for children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to FYCOMPA (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS. Finally, on July 18, 2023, Catalyst acquired an exclusive license for North America for AGAMREE (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne Muscular Dystrophy.  AGAMREE previously received FDA Orphan Drug and Fast Track designations and was approved for commercialization in the U.S. on October 26, 2023.

For more information about Catalyst Pharmaceuticals, Inc., please visit the Company's website at www.catalystpharma.com. For Full Prescribing and Safety Information for FIRDAPSE, please visit www.firdapse.com. For Full Prescribing Information, including Boxed WARNING for FYCOMPA, please visit www.fycompa.com. For Full Prescribing Information for AGAMREE, please visit www.agamree.com.