The US Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). It is expected that the administration will complete its review of the application on October 26, 2023. If approved, the treatment is expected to be launched in the US by the end of 2023.
“We are delighted that the FDA has accepted Santhera’s vamorolone NDA for filing,” Dario Eklund, the chief executive officer of Santhera said in a press release.
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“We believe this product addresses a clear unmet medical need and for Santhera, this represents the achievement of an important milestone with key significance for our future success. We look forward to working closely with US regulators to advance vamorolone towards approval,” the researchers continued.
Eric Hoffman, PhD, the president and chief executive officer of ReveraGen BioPharma, the co-developers of the treatment added, “This is an exciting time for the Duchenne community as the data generated across the clinical trial program indicate that vamorolone has the potential to address relevant aspects in patient care that could also enhance treatment outcomes. If approved, vamorolone will emerge as an additional treatment option in current standards of care in DMD, with the potential to address unmet medical needs and treat a majority of Duchenne patients starting at an early age.”
Vamorolone is an experimental synthetic steroid that binds to the same receptor as corticosteroids, the current standard of care for DMD. However, its downstream activity is different, and it is therefore considered “a dissociative antiinflammatory” as efficacy and safety concerns are dissociated.
The treatment was shown to be safe and well-tolerated in clinical trials and met its primary endpoint of time to stand velocity compared to a placebo in a pivotal study.
A marketing authorization application for vamorolone is also under review by the European Medicines Agency.
Santhera and ReveraGen announce FDA acceptance of New Drug Application for vamorolone in Duchenne muscular dystrophy. News release. Santhera. January 9, 2023.