Santhera Pharmaceuticals announced that Swissmedic, the Swiss Agency for Therapeutic Products, has accepted for review the marketing authorization application (MAA) for AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD). This follows approvals in the U.S., EU, and UK, and acceptance for priority review in China. The submission aims to address the high unmet need for improved treatment options for DMD patients in Switzerland.
Regulatory Pathway
Santhera requested Swissmedic to assess AGAMREE® under Article 13 TPA, allowing consideration of foreign regulatory authorities’ assessments, specifically the EU approval from December 2023. This approach can expedite the approval process by leveraging existing evaluations from countries with comparable regulatory standards. The outcome of the Swissmedic review is anticipated in late H1-2026, potentially accelerating to early 2026 if Article 13 is accepted. Approval in Switzerland, alongside EMA/FDA/MHRA approvals, could facilitate access to other markets through abbreviated reviews.
AGAMREE®: A Dissociative Steroid
AGAMREE® (vamorolone) is a novel drug that binds to the same receptor as glucocorticoids but modifies its downstream activity. It is not a substrate for 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes, which can cause local drug amplification and corticosteroid-associated toxicity. This mechanism aims to dissociate efficacy from steroid safety concerns, positioning AGAMREE® as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care for children and adolescents with DMD.
Clinical Trial Data
In the pivotal VISION-DMD study, AGAMREE® met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment, demonstrating a favorable safety and tolerability profile. Common side effects included cushingoid features, vomiting, weight increase, and irritability, generally mild to moderate in severity. Data suggest that AGAMREE®, unlike corticosteroids, does not restrict growth and has no negative effects on bone metabolism, as indicated by normal bone formation and resorption serum markers.
Duchenne Muscular Dystrophy (DMD)
DMD is a rare, inherited X-chromosome-linked disease primarily affecting males. It is characterized by inflammation leading to muscle fibrosis, progressive muscle degeneration, and weakness. Key milestones include loss of ambulation, self-feeding, the need for assisted ventilation, and the development of cardiomyopathy. DMD typically reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care.
Shabir Hasham, MD, Chief Medical Officer of Santhera, commented, “Submission of AGAMREE for DMD to Swissmedic is an important milestone, following approvals from the U.S. FDA, the European Commission in the EU, and the UK MHRA, in addition to acceptance for priority review from China’s NMPA. We look forward to working closely with Swissmedic to address the high unmet need for improved treatment options for patients suffering from DMD in Switzerland.”
