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.
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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for Agamree on 16 January 2025, following confirmation that no appeals were submitted against its December 2024 final draft guidance.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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By GlobalDataAgamree received UK regulatory approval in January 2024, building on similar decisions by the US Food and Drug Administration (FDA) and European Commission (EC) in October 2023 and December 2023, respectively. While it is approved in the same indication in Europe, patients as young as two years old in the US have access to the treatment. Santhera sold US commercialisation rights for Agamree to Catalyst Pharmaceuticals in July 2023 in a deal worth up to $231m.
According to GlobalData’s Pharma Intelligence Center, Agamree is forecast to generate up to $373m in 2030.
GlobalData is the parent company of Pharmaceutical Technology.
Other approved treatments for DMD include Sarepta’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl). Initially approved via the accelerated pathway in 2023 for ambulatory boys aged between four and five years, Elevidys’ label was expanded in June 2024 to all DMD patients over four years of age with confirmed DMD mutations.
Among others in the same space, US company Regenxbio is also planning a biologics licence application (BLA) filing in 2026 for RGX-202, its investigational DMD gene therapy, following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial (NCT05693142).
