Catalyst Pharmaceuticals to Present at MDA Clinical & Scientific Conference Details of Registry for Study of Long-Term Safety and Quality of Life in Duchenne Muscular Dystrophy Patients Treated with AGAMREE - Seite 2

AGAMREE was granted U.S. FDA approval on October 26, 2023, and was granted Orphan Drug and Rare Pediatric Disease designation status for DMD in the U.S., making it eligible for seven years of orphan drug exclusivity upon approval. AGAMREE also has issued and pending patents that could provide protection until 2040. In Europe, it has received Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.

References:
[1] Dang UJ et al. (2024) Neurology 024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.
[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
[3] Liu X et al. (2020). Proc Natl Acad Sci USA 117:24285-24293
[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508
[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.
[6] Hasham et al., MDA 2022 Poster presentation. Link.
[7] Applicable drug labeling: Summary of Product Characteristics (SmPC). English.

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 by the FDA for commercialization in the U.S. on October 26, 2023.