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FDA receives Santhera and ReveraGen’s application for Duchenne muscular dystrophy treatment

The submission includes positive data from the pivotal phase 2b and four open-label studies

FDA

The US Food and Drug Administration (FDA) has received Santhera Pharmaceuticals (Santhera) and ReveraGen BioPharma’s completed rolling submission of a New Drug Application (NDA) for the priority review of vamorolone in the treatment of Duchenne muscular dystrophy (DMD), the companies announced.

DMD is a rare inherited X-chromosome-linked disease, almost exclusively affecting males, and is caused by a change or mutation in the gene that encodes instructions for dystrophin, meaning those with the condition are unable to adequately make the micro-dystrophin protein on their own.

Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Patients typically go on to experience increasing difficulty in breathing and cardiac dysfunction, which is usually fatal.

“If approved, vamorolone will emerge as an addition to 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,” said Eric Hoffman, president and chief executive officer of ReveraGen BioPharma.

The NDA submission for vamorolone is supported by positive data from the pivotal phase 2b VISION-DMD study which comprised a 24-week period to demonstrate efficacy and safety of vamorolone at 2 and 6mg/kg a day versus prednisone and placebo, followed by a 24-week period to evaluate the maintenance of efficacy and collect additional longer-term safety and tolerability data.

The study met the primary endpoint time to stand velocity versus placebo at 24-weeks and showed a good safety and tolerability profile, the companies reported.

The filings also include data from four open-label studies, including extension, in which vamorolone was administered at doses between 2 and 6mg/kg a day for a total treatment period of up to 30 months and an external comparator study.

“Completion of the vamorolone NDA submission is a major step towards our goal of bringing this investigational therapy to patients living with DMD, and represents a vital milestone for Santhera,” said Dario Eklund, chief executive officer of Santhera.

In Europe, Santhera has submitted a Marketing Authorisation Application to the European Medicines Agency in September 2022, seeking approval for vamorolone in the EU, as well as in Norway, Liechtenstein and Iceland.

Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD, and has received Fast Track and Rare Paediatric Disease designations by the FDA, as well as Promising Innovative Medicine status from the UK Medicines and Healthcare products Regulatory Agency for DMD.

Article by
Emily Kimber

31st October 2022

From: Regulatory

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