Santhera Pharmaceuticals announces that AGAMREE (vamorolone) has been approved in the European Union (EU) for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older, independent of the underlying mutation and ambulatory status.
The European Medicines Agency (EMA) acknowledged clinically important safety benefits of AGAMREE with regards to maintaining normal bone metabolism, density and growth compared with 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.
This makes AGAMREE the first and only medicinal product to have received full approval in the EU and, following US Food and Drug Administration (FDA) approval in October, the first authorised treatment in both the US and EU to treat patients with DMD.
“We are thrilled to receive approval from the EC to bring AGAMREE to patients in the EU, which highlights the favourable safety and tolerability profile compared with conventional corticosteroids, including benefits for bone health and growth,” said Dario Eklund, CEO of Santhera.
“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.”
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 doses between 2 and 6 mg/kg/day 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 a placebo, whereas 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.
Unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism as measured by bone biomarkers, nor in a significant reduction of bone mineralisation in the spine as measured by Dual Energy X-Ray Absorptiometry (DXA) after 48 weeks in the clinical studies.
Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.
