FDA approval Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals
Source: GlobeNewswire Inc.
Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX) today reported that Santhera Pharmaceuticals ("Santhera") has obtained U.S. Food and Drug Administration ("FDA") approval for AGAMREE® (vamorolone) oral suspension 40 mg/mL for use in treating Duchenne Muscular Dystrophy ("DMD") in patients aged two years and older. AGAMREE offers a novel corticosteroid treatment option for DMD, addressing a significant unmet medical need. In July 2023, Catalyst secured the exclusive North American license and commercial rights for AGAMREE from Santhera for DMD and other potential indications, bolstering its neuroscience commercial portfolio with a highly synergistic neuromuscular asset. As part of that transaction, Santhera will promptly transfer the approved New Drug Application for AGAMREE to Catalyst.
FDA’s approval of AGAMREE® was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, AGAMREE was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard-of-care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory, and behavior.
“We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with Duchenne Muscular Dystrophy and potentially other chronic inflammatory diseases. The approval of AGAMREE underscores the potential of reshaping the DMD treatment paradigm for this life-threatening rare disease. The addition of AGAMREE expands our rare neuromuscular disease portfolio, and we look forward to executing on our proven commercial capabilities to bolster our long-term growth potential,” said Patrick J. McEnany, Chairman and CEO of Catalyst Pharmaceuticals. “Our unwavering commitment extends beyond this important milestone as we are resolute in our mission to ensure that DMD patients in the U.S. have access to this treatment option as we believe that AGAMREE may offer the potential of increasing the duration of ambulation and mobility in these patients, thereby significantly improving their overall quality of life. We expect to launch the product in the first quarter of 2024. At that time, we will introduce a comprehensive financial assistance program aimed at helping ensure accessibility and minimizing patient co-pays and deductibles, thereby enhancing affordability for all DMD patients. We look forward to successfully commercializing this product with a continued commitment to serving our patient communities."
Upon the transfer of AGAMREE's NDA into its neuromuscular franchise, Catalyst will harness its product portfolio synergies by leveraging its well-established expertise and proven commercial capabilities. The Company plans to launch the product in Q1 2024, spearheaded by its seasoned and experienced U.S. commercial and medical affairs neuromuscular teams.
AGAMREE was granted Orphan Drug and Rare Pediatric Disease designations status for DMD in the U.S. and will be eligible for seven years of orphan drug exclusivity upon approval date and has issued pending patents that could provide protection until 2040.
