Santhera and ReveraGen Start Rolling NDA Submission to the FDA for Vamorolone for the Treatment of Duchenne Muscular Dystrophy

Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc (US: private) have announced the initiation of a rolling new drug application (NDA) submission to the U.S. Food and Drug Administration (FDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). Vamorolone for DMD has been granted Fast Track Designation by the FDA.

Santhera and ReveraGen have now commenced the NDA filing as a rolling submission following a successful pre-NDA meeting with the FDA in November 2021. In its conclusions from this meeting, the FDA considered the proposed clinical efficacy and safety data sufficient to support an NDA filing of vamorolone for the treatment of DMD. Acceptance of the NDA will be subject to FDA’s review of the complete filing, the companies said.

“The NDA filing for vamorolone marks a tremendous milestone for Santhera and an important next step for the Duchenne community. The potential benefits of vamorolone could address significant unmet needs that represent a burden to DMD patients and their families,” said Dario Eklund, Chief Executive Officer of Santhera. “We are fully focused on working closely with the FDA and completing the submission as soon as possible.”

“We are delighted about the initiation of the filing for approval for vamorolone as it represents a culmination of over a decade of scientific research for the benefit of patients with DMD. In having granted Fast Track Designation, the FDA has acknowledged the significant innovation vamorolone could bring in addressing a high unmet medical need of patients with DMD,” said Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma. “We thank study participants, their families and caregivers, as well as investigators and study personnel, for their commitment to the vamorolone program.”

Santhera and ReveraGen expect to complete the NDA filing in the second quarter of 2022. Based on FDA review timelines, notification from the FDA on the acceptance of the filing for review is expected in August 2022. In its assessment, the FDA will also determine eligibility of vamorolone for priority review which Santhera and ReveraGen will request upon completion of the rolling NDA submission. If granted, this would shorten review time and set an anticipated approval date for as early as the first quarter of 2023. Subject to FDA approval, Santhera plans to launch vamorolone in the U.S., the first country, shortly thereafter with its own organization, the companies said.

In Europe, Santhera plans to submit a marketing authorization application (MAA) for vamorolone for the treatment of DMD to the European Medicines Agency (EMA) in Q3-2022. Assuming a review time of about one year, this could pave the way for approval and launch in H2-2023. Santhera expects the first European launch country to be Germany.

Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.

Vamorolone was discovered by US-based ReveraGen and is being developed in collaboration with Santhera who owns worldwide rights to the drug candidate for all indications. In January 2022, Santhera licensed rights to vamorolone in rare diseases for the Greater China region to Sperogenix Therapeutics.