Santhera’s AGAMREE receives FDA approval for Duchenne muscular dystrophy treatment

The U.S. FDA has given the green light for Santhera’s vamorolone-based treatment, AGAMREE, against Duchenne muscular dystrophy (DMD). This approval presents a significant milestone for the company from Basel-Landschaft. AGAMREE serves as an effective alternative to standard corticosteroids, which were the drug of choice against DMD until now. While causing less adverse effects, AGAMREE has shown promise on slowing disease progression.

Upon FDA approval, U.S. license holder Catalyst Pharmaceuticals will pay USD 36 million to Santhera, of which USD 26 million are used by Santhera to cover their contracted third-party milestone obligations, while USD 10 million are an approval milestone for the company. The agreement between the two pharmaceutical companies also includes a payment of up to USD 105 million and up to low-teen percentage royalties from Catalyst to Santhera for sales-based milestones. The commercialization of AGAMREE in the U.S. will be launched in Q1 of 2024.

The FDA’s decision to approve AGAMREE was based on the critical Phase 2b VISION-DMD study, which demonstrated vamorolone’s efficacy compared to other corticosteroids, with less side effects related to bone health, growth trajectory and behavior.

DMD is a rare X-chromosome-linked inheritable disease, which almost exclusively affects boys and men. The disease is characterized by inflammation (at birth or shortly thereafter), which leads to progressive muscle degeneration and weakness. Patients’ life expectancy is reduced to before the fourth decade due to respiratory and/or cardiac failure. Less than 10 cases of DMD occur in every group of 100,000 people assigned male at birth globally. The DMD community and healthcare practitioners are very hopeful about this development. Pat Furlong, Founding President & CEO of Parent Project Muscular Dystrophy (PPMD), emphasized the significance of AGAMREE as a treatment with a better tolerability profile, addressing a pressing unmet medical need for DMD patients.

On top of the FDA approval, AGRAMEE is poised for approval in the European Union by the European Commission (EC) in late 2023, thus becoming the first fully approved DMD treatment in both the U.S. and Europe, strengthening Santhera’s position in the field of rare diseases.

(Press release / SR)