Santhera Pharmaceuticals (SIX:SANN, OTC:SPHDF, FRA:S3F0), the Swiss rare disease specialist, has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommending that its Duchenne muscular dystrophy (DMD) treatment AGAMREE be approved for children from two years of age.
DMD is a severe inherited muscle-wasting disease that progressively weakens skeletal, heart, and respiratory muscles, typically affecting boys from early childhood.
AGAMREE, known generically as vamorolone, is currently authorised in the European Union for DMD patients aged four and older, and the CHMP opinion, if confirmed by the European Commission, would extend that label to include two and three-year-olds, a group where early anti-inflammatory intervention may offer the greatest long-term benefit.
Chief executive Dario Eklund said the opinion marked an important step towards making AGAMREE available to every DMD patient who could benefit, noting that decisions around treatment initiation in very young children are particularly challenging given the limited options available.
AGAMREE is a dissociative corticosteroid, meaning it is designed to deliver anti-inflammatory efficacy while reducing the systemic side effects associated with conventional long-term steroid therapy, which remain a significant concern in young patients.
Long-term data spanning up to eight years of treatment, presented at the Muscular Dystrophy Association Clinical and Scientific Conference in March 2026, showed that AGAMREE produced a markedly lower incidence of vertebral fractures compared with the standard corticosteroid deflazacort, at 8.1% versus 41.9%.
Treated patients also maintained a normal growth trajectory, with a mean height advantage of more than 12 centimetres over those on conventional corticosteroids, and a lower rate of cataracts, with no observed cases of glaucoma.
Chief medical officer Shabir Hasham said initiating anti-inflammatory therapy early in DMD had the potential to reshape the long-term disease trajectory, and that the CHMP opinion reflected the strength of AGAMREE's clinical and safety data.
AGAMREE is already approved in the US, UK, Switzerland, China, Canada, and Hong Kong, with regional licensing partners covering North America, Asia, and Australasia.