EU approval for Santhera's vamorolone in sight

A marketing authorization decision from the European Commission is expected within approximately two months of the positive CHMP opinion. Subject to approval, AGAMREE (the brand name for vamorolone) will be the first and only medicinal product fully approved by the EMA for the treatment of DMD and the marketing authorization will be valid in all 27 member states of the European Union as well as Iceland, Liechtenstein and Norway. Potential launches of AGAMREE in the EU, with Germany taking the lead, are planned to start in Q1-2024.  

Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. Currently available data show that vamorolone, unlike corticosteroids, has no restriction of growth and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers.

In the U.S., FDA established the target Prescription Drug User Fee Act (PDUFA) action date for its regulatory decision on the vamorolone NDA as October 26, 2023. Subject to approval, Santhera’s licensing partner Catalyst Pharmaceuticals plans to launch vamorolone in the U.S. in Q1-2024.

“We are thrilled about the CHMP’s positive opinion, which recognizes the urgent medical need for an effective and well tolerated treatment for this devastating disease. We can now execute on our plans to ensure AGAMREE is made available to patients in the EU as soon as the European Commission marketing authorization is received,” said Dario Eklund, CEO of Santhera. “The recommendation by the CHMP is a testament to the dedication and collaboration of all those involved in the development of vamorolone, including ReveraGen Biopharma, the DMD patient community, researchers, healthcare professionals, and our employees.”

(Press release / SK)