Santhera gains Positive NICE Guidance for AGAMREE® in treating Duchenne Muscular Dystrophy

Santhera Pharmaceuticals  has announced a significant milestone with the National Institute for Health and Care Excellence (NICE) issuing positive Final Guidance recommending AGAMREE® (vamorolone) for use in the National Health Service (NHS) in England, Wales, and Northern Ireland. The recommendation covers the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

The approval follows the confirmation that no appeals were lodged against the Final Draft Guidance (FDG) recommendation issued on December 10, 2024. This landmark decision paves the way for Santhera to initiate launch preparations for AGAMREE in the UK, with first sales expected in the current quarter.

AGAMREE is distinguished as the first and only medicinal product for DMD to achieve full regulatory approval across the European Union, the United States, and the United Kingdom. Its approval by the Medicines and Healthcare products Regulatory Agency (MHRA) on January 11, 2024, builds on earlier European Medicines Agency (EMA) approval, recognizing its clinically significant benefits in tolerability.

Compared to traditional corticosteroids, AGAMREE has shown superior tolerability by maintaining normal bone metabolism, density, and growth, while delivering comparable efficacy in clinical trials. This balance offers a critical advantage for managing the progressive muscle-wasting disease, which primarily affects young boys.

Santhera’s achievement reflects its commitment to improving the standard of care for DMD patients, providing a new option that addresses the unmet needs in this challenging condition, and marks a new era in its treatment landscape.