Santhera Pharmaceuticals and ReveraGen BioPharma have announced the successful completion of the mid-cycle review meeting by the U.S. Food and Drug Administration (FDA) of the new drug application (NDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).

At the recent mid-cycle review meeting, the FDA indicated that no significant review or safety concerns were noted up to that point in their ongoing review. The FDA also reaffirmed its earlier decision to forgo an Advisory Committee Meeting. In addition, the FDA confirmed the PDUFA (Prescription Drug User Fee Act) date of October 26, 2023, on which an approval decision on the vamorolone NDA is expected.

“We are very pleased to have completed this stage of the review process. We look forward to working closely with the FDA during the remainder of the review process and are confident about being able to provide an emerging therapy to patients with DMD in the U.S. soon.”

Shabir Hasham, MD, Chief Medical Officer of Santhera

“The progress we have made and the positive interactions with the FDA to date give us a real sense of accomplishment. Step by step we have come even closer to bringing a novel therapy to patients that, if approved, would contribute to improving the current standard of care in DMD.”

Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma

With regards to launch plans for vamorolone:

In Europe, the review of the marketing authorization application (MAA) for vamorolone by the European Medicines Agency (EMA) is on track. A CHMP opinion is expected in Q4-2023, followed by an approval decision by the European Commission (EC) in late 2023.

In the UK, a corresponding MAA is under review by the Medicines and Healthcare Products Regulatory Agency (MHRA). Subject to approvals, Santhera plans to launch vamorolone in both the U.S. and the EU in late 2023.

Vamorolone has been granted Orphan Drug status for DMD in the U.S. and Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.

As an investor in vamorolone, Defeat Duchenne Canada is working with these companies to better understand the launch plans in Canada.