Santhera Pharmaceuticals has announced the introduction of its AGAMREE (vamorolone) to treat Duchenne muscular dystrophy (DMD) patients aged four years and above in Germany.
This marks a significant milestone as AGAMREE becomes the first medicinal asset for DMD to receive full approval in the EU.
The development follows the European Commission’s approval on 18 December 2023. AGAMREE is now authorised for use in all 27 EU member states along with Liechtenstein, Iceland, Norway, the UK and the US.
The approval by the European Medicines Agency highlighted the safety benefits of AGAMREE in maintaining normal bone metabolism, density and growth versus conventional corticosteroids.
Patients treated with AGAMREE or placebo exhibited normal and comparable growth, while growth stunting was seen in children who received prednisone.
Patients transitioning from standard corticosteroids to AGAMREE continued to experience efficacy benefits and recovery in growth and bone health.
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The active ingredient in AGAMREE is vamorolone, a potential substitute for currently available corticosteroids for children and adolescents with DMD.
In the VISION-DMD study, vamorolone met the primary endpoint of Time to Stand velocity compared with placebo at 24 weeks, demonstrating a robust safety and tolerability profile.
Cushingoid features [the constellation of symptoms and signs caused by an excess of the hormone cortisol, also known as Cushing syndrome], vomiting and vitamin D deficiency were the most frequently seen adverse events in the trial.
Santhera Pharmaceuticals CEO Dario Eklund stated: “We are very happy that AGAMREE is now commercially available in Germany, the first country worldwide, for the treatment of DMD.
“This significant milestone represents Santhera’s commitment to fill the unmet need in DMD and provide a safe and effective treatment for DMD patients. For Santhera, this launch signifies a leap forward as the company enters the commercial stage.
“In parallel, we continue to work towards our own staged commercial rollout in the large European markets, alongside ongoing discussions with distribution partners for other regions.”
