RARE Daily

EU Approves Santhera’s DMD Therapy Agamree

December 20, 2023

Rare Daily Staff

The European Union approved Santhera Pharmaceuticals’ Agamree for the treatment of the rare, neuromuscular condition Duchenne muscular dystrophy.

The approval of Agamree is for use in patients 4 years of age and older, independent of the underlying mutation and ambulatory status.

Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 patients in the United States are affected by DMD, with approximately 70 percent of patients currently receiving concomitant corticosteroid treatment.

Agamree’s (vamorolone) mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity and, as such, is considered a novel corticosteroid with dissociative properties in maintaining efficacy, with a better-tolerated side effect profile. The European Medicines Agency noted clinically important safety benefits of Agamree with regards to maintaining normal bone metabolism, density, and growth compared to standard of care corticosteroids, alongside similar efficacy. In addition, patients who switched from a standard of care corticosteroid to Agamree maintained the efficacy benefit while recovering their growth and bone health.

The approval by the EC was based on data from the positive pivotal VISION-DMD study and three open- label studies in which vamorolone was administered at multiple doses for a total treatment period of up to 30 months. In the pivotal VISION-DMD study, boys treated with vamorolone on average maintained growth similar to those treated with placebo, while those treated with prednisone on average experienced growth stunting. Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study.

The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

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.

Agamree has Orphan Drug status for DMD in the United States and in Europe and has received Fast Track and Rare Pediatric Disease designations by the FDA. Vamorolone is approved for use in the United States and the European Union.

“Our team is now focused on ensuring Agamree is made available to the Duchenne patients as soon as possible, with a first commercial launch planned for Germany in Q1-2024,” said Dario Eklund, CEO of Santhera.

Photo: Dario Eklund, CEO of Santhera

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