Core Insights - Catalyst Pharmaceuticals has announced that Health Canada accepted the New Drug Submission for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), which has been granted Priority Review, potentially leading to marketing authorization by the end of 2025 [1][2] - If approved, AGAMREE would be the first and only treatment option for DMD patients in Canada, addressing a significant unmet medical need [2] - AGAMREE received FDA approval on October 26, 2023, and became commercially available in the U.S. on March 13, 2024 [2] Company Overview - Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing novel medicines for rare diseases [1][7] - The company has a commitment to patient care and accessibility, ensuring that patients receive necessary treatments through comprehensive support services [7] - Catalyst has been recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's Fastest-Growing Companies [7] Product Information - AGAMREE (vamorolone) is designed to have a unique mode of action that differentiates its effects on glucocorticoid and mineralocorticoid receptors, potentially offering a better-tolerated side effect profile compared to traditional corticosteroids [4] - In the pivotal VISION-DMD study, AGAMREE met its primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks, demonstrating good safety and tolerability [4] Market Context - Duchenne muscular dystrophy (DMD) is a genetic disorder that primarily affects males, leading to progressive muscle degeneration and weakness, with symptoms typically appearing in early childhood [3] - The condition is caused by mutations in the dystrophin gene, which is crucial for muscle fiber integrity [3] - Current treatment options for DMD are limited, highlighting the importance of AGAMREE as a potential new therapy [2][3] Collaboration - Kye Pharmaceuticals, Catalyst's sub-licensee in Canada, is responsible for the marketing of AGAMREE and is also involved in the commercialization of Catalyst's flagship product, FIRDAPSE®, for Lambert-Eaton myasthenic syndrome [2][5] - Kye Pharmaceuticals focuses on bringing novel prescription medicines to the Canadian market, addressing clinically significant unmet needs across various therapeutic areas [5]