Swiss biotech Santhera Pharmaceuticals has started launch preparations for its Duchenne muscular dystrophy (DMD) drug Agamree (vamorolone) in the UK, following a recommendation from England’s price watchdog.
Approved for use in the National Health Service (NHS) in England, Wales, and Northern Ireland, Agamree is indicated for patients aged four years and older with DMD. The company expects the first sales to commence within this quarter.
DMD is a rare progressive genetic disorder that primarily affects boys. It causes muscle weakness and degeneration, leading to loss of mobility and premature death. Standard treatment typically involves corticosteroids to manage inflammation and preserve muscle strength, but these therapies are associated with significant side effects like growth stunting and bone health issues.
England’s National Institute for Health and Care Excellence (NICE) issued a positive final guidance for Agamree on 16 January 2025, following confirmation that no appeals were submitted against its December 2024 final draft guidance. This represents a reversal of NICE’s initial decision in March 2024, when the agency declined to recommend the drug due to concerns about its cost-effectiveness compared to standard-of-care corticosteroids. Subsequent submissions from Santhera addressed these issues, with NICE acknowledging that Agamree may offer fewer side effects than current options.
Agamree is a first-in-class anti-inflammatory drug that binds to the same receptor as glucocorticoids but modulates its downstream activity differently, reducing the risk of corticosteroid-associated toxicities. The drug was designed as an alternative to traditional corticosteroids to mitigate side effects such as stunted growth and bone metabolism issues.
The pivotal VISION-DMD study (NCT03439670) demonstrated that boys treated with Agamree maintained normal bone growth patterns and showed no signs of reduced bone metabolism or mineralisation after 48 weeks, unlike those treated with the corticosteroid prednisone. Patients transitioning from corticosteroids to Agamree reportedly recovered growth and bone health while maintaining efficacy.
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