CHMP recognizes urgent medical need for treating Duchenne patients

CHMP has adopted a positive opinion in favor of approval of a Swiss specialty pharmaceutical company Santhera Pharmaceuticals’ AGAMREE (vamorolone) for the treatment of Duchenne Muscular Dystrophy patients aged 4 years and older.

In its recommendation for approval, the CHMP acknowledges the positive benefit-risk profile of AGAMREE in this patient population, including certain safety benefits of AGAMREE compared to standard of care corticosteroids in the treatment of DMD, Santhera said in its press release Friday. The company also said it plans for a first commercial launch in Germany in Q1-2024, subject to approval by the EC.

“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.”

“We are very pleased about the positive vote of the European Medicines Agency. This offers Duchenne patients a new treatment alternative to slow down the progression of the disease with fewer side effects,” said Maggie C. Walter, MD, MA, Associate Professor for Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University of Munich, Germany.

Clinical evidence

The clinical evidence for the efficacy and safety of AGAMREE (vamorolone) in the regulatory submission was derived from the positive pivotal VISION-DMD study and three open-label studies (including extensions), in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months, as well as an external comparator study (FOR-DMD [6]) and several clinical pharmacology studies, Santhera said in the press release. The studies were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.