CORAL GABLES, Fla. - Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX), a biopharmaceutical company, has announced the commercial launch of AGAMREE (vamorolone) for Duchenne Muscular Dystrophy (DMD) treatment in patients aged two years and older in the United States. The U.S. Food and Drug Administration (FDA) approved AGAMREE on October 26, 2023, and it is now available by prescription.
AGAMREE, an oral suspension 40 mg/mL, has been introduced as an alternative steroid treatment for DMD, a rare and life-threatening neuromuscular disorder. Richard J. Daly, CEO of Catalyst, highlighted that AGAMREE could offer improved quality of life with fewer side effects compared to current corticosteroid treatments. The company has also established the Catalyst Pathways® Program to provide personalized support and financial assistance to ensure affordable access to AGAMREE for DMD patients.
The FDA approval of AGAMREE was based on the results from the Phase 2b VISION-DMD study, which showed that the treatment met its primary endpoint, Time to Stand (TTSTAND) velocity, and exhibited a safety profile with reduced adverse events related to bone health and growth. The most common side effects reported were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency, generally of mild to moderate severity.
Catalyst Pharmaceuticals has been granted Orphan Drug Exclusivity (ODE) and New Chemical Entity Exclusivity (NCE) for AGAMREE in the U.S., providing seven and five years of market exclusivity, respectively, from the approval date. The drug also has pending patents that could extend protection until 2040.
DMD affects an estimated 11,000 to 13,000 patients in the U.S., with about 70% of them currently receiving corticosteroid treatment. AGAMREE's mode of action is considered to have dissociative properties, potentially offering a more tolerable side effect profile while maintaining efficacy.
This news is based on a press release statement from Catalyst Pharmaceuticals.
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