Santhera’s Vamorolone for Duchenne Muscular Dystrophy (DMD) Approved in the European Union

Pratteln, Switzerland – The European Commission (EC) granted marketing authorization to vamorolone for the treatment of patients with Duchenne muscular dystrophy (DMD), who are 4 years of age and above, according to a press release from Santhera, the developers of the treatment.

Vamorolone will be sold under the brand name Agamree^® and is expected to be commercially available in Germany in the first quarter of 2024.

“We are thrilled to receive approval from the EC to bring AGAMREE to patients in the EU, which highlights the favorable safety and tolerability profile over conventional corticosteroids, including benefits for bone health and growth,” said Dario Eklund, the chief executive officer of Santhera, in the press release.

The approval is based on results from a pivotal clinical trial called VISION-DMD and 3 open-label trials, which showed that patients with DMD treated with vamorolone had similar growth to those given a placebo. In contrast, patients treated with the corticosteroid drug prednisone had growth stunting.

Moreover, patients who switched from prednisone to vamorolone after 24 weeks resumed their growth.

Finally, vamorolone treatment did not lead to a reduction in bone metabolism or bone mineralization in the spine after 48 weeks.

Vamorolone is a novel drug, which binds to the same receptor as glucocorticoids. However, it modifies the downstream activity of the receptor instead of acting as a substrate for the 11-β-hydroxysteroid dehydrogenase enzymes that may be responsible for local tissue amplification and corticosteroid-associated toxicity. It is, therefore, an alternative to corticosteroids, which are currently the standard of care for patients with DMD.

Valmolorone is also approved in the US and is now the only treatment for patients with DMD approved in both the US and the EU.

DMD is a rare genetic disease caused by a mutation in the DMD gene. This gene codes for a protein essential for muscle health, so in its absence, muscles become damaged and are replaced by scar tissue over time.