Santhera Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved AGAMREE® (vamorolone) oral suspension 40 mg/ml for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. The FDA 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. The studies in the development program were carried out by Santhera?s partner ReveraGen and 32 academic clinical trial centers in 11 countries. With the FDA approval of AGAMREE for DMD, Catalyst will pay USD 36 million to Santhera, consisting of a USD 10 million approval milestone to the Company and an additional USD 26 million to cover contracted third-party milestone obligations.

Furthermore, under the terms of the agreement, Catalyst will pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties, and will assume Santhera's corresponding third-party royalty obligations on vamorolone sales in all indications in North America. Santhera will now transfer the U.S. marketing authorization (NDA) for AGAMREE to its partner Catalyst Pharmaceuticals who holds an exclusive license for AGAMREE in North America and plans to launch the product in the U.S. in First Quarter -2024. In Europe, following the positive opinion from the CHMP received on October 12, 2023, approval of AGAMREE (vamorolone) for the treatment of DMD by the European Commission is expected in late 2023.

Subject to approval, AGAMREE will be the only medicinal product fully approved in both territories, the EU and the U.S., for the treatment of DMD.