Santhera Pharmaceuticals (SANN) has received a positive opinion from the European Medicines Agency's CHMP to expand the use of AGAMREE (vamorolone) to children with Duchenne muscular dystrophy as young as two years old, a step that could widen access to treatment at an earlier stage of the disease.
Duchenne muscular dystrophy (DMD) is a rare progressive muscle-wasting disorder that typically begins in early childhood. Current treatment options often rely on conventional corticosteroids, which can help slow disease progression but are associated with long-term side effects that are especially challenging in very young children. AGAMREE, a dissociative corticosteroid, is already approved in the EU for patients aged four and older.
A Step toward earlier intervention
If the European Commission confirms the CHMP recommendation, AGAMREE's authorized age range would expand to include children from two years of age. According to Santhera, earlier anti-inflammatory treatment may offer long-term benefit, particularly in a population where tolerability concerns with standard corticosteroid remain significant.
Santehra CEO Dario Eklund said the opinion represents an important milestone toward making the therapy available to all eligible DMD patients. The company noted that the treatment decisions in very young children can be difficult due to limited options and the burden of side effects associated with existing therapies.
Clinical data supporting the decision
AGAMREE's profile is based on its selective glucocorticoid-receptor activity, designed to maintain anti-inflammatory effects while reducing some of the systemic impacts seen with reducing some of the systemic impacts seen with traditional corticosteroids. In the pivotal Phase 2b VISION-DMD study, the therapy met its primary endpoint, showing a statistically significant improvement in Time to Stand velocity at 24 weeks compared with placebo.
Long-term data presented at the 2025 MDA Clinical & Scientific Conference showed sustained efficacy and a differentiated safety profile over several years of treatment, including lower rates of vertebral fractures and cataracts compared with standard corticosteroids.
About AGAMREE
AGAMREE is approved for DMD in multiple regions, including the U.S., EU, UK, Switzerland, China, Hong Kong, and Canada. Santhera holds global rights to the drug and has out-licensed commercialization in several territories, including North America, China, and Japan.
SANN has traded between CHF 9.38 and CHF 18.26 over the past year. The stock is currently trading at CHF 17.26, up 1.89%.
For comments and feedback contact: editorial@rttnews.com
Business News
June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.