FDA Completes Mid-Cycle Review Meeting for Vamorolone NDA – What Does This Mean?

Santhera Pharmaceuticals and ReveraGen BioPharma, Inc. have just received the green light from the U.S. Food and Drug Administration for the new drug application of vamorolone, a potential treatment for Duchenne muscular dystrophy. After a successful mid-cycle review meeting, the two companies are now one step closer to helping those affected by this debilitating disease.

At the mid-cycle review meeting, the FDA provided positive feedback with no significant review or safety concerns noted. The FDA confirmed that no Advisory Committee Meeting was needed and set the PDUFA date of October 26, 2023, for a final approval decision on the vamorolone NDA. With the FDA’s encouraging review, vamorolone is on its way to potentially becoming a promising new treatment option.

The FDA has been closely reviewing the NDA and has so far conducted successful inspections of the contract manufacturer, the sponsor, and several clinical trial sites. All the inspections resulted in satisfactory outcomes.

Santhera is thrilled to have completed the review process for a potential emerging therapy for patients with DMD. We are excited to continue working with the FDA during the review process and are confident that we will be able to provide this therapy to patients in the U.S. soon. Shabir Hasham, MD, Chief Medical Officer of Santhera, is eager for the positive outcome of this endeavor.

At ReveraGen BioPharma, progress has been made and positive interactions with the FDA have been achieved, giving the company a sense of accomplishment. If approved, their novel therapy could potentially lead to a much-needed improvement in the current standard of care for patients with DMD. Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma, is confident that every step taken brings them closer to making this a reality.

Santhera is on track to launch vamorolone in the European Union and the United States by late 2023. The review of the marketing authorization application (MAA) for vamorolone by the European Medicines Agency (EMA) is underway, with a CHMP opinion expected in the fourth quarter of 2023.

The Medicines and Healthcare products Regulatory Agency (MHRA) is also conducting a review of the MAA in the UK. If all goes according to plan, Santhera can look forward to offering vamorolone to European and American consumers in the near future.

Vamorolone has been given the green light to pursue its mission of helping those with Duchenne Muscular Dystrophy (DMD) by being granted Orphan Drug status in the U.S. and Europe, as well as Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA. This is an exciting development for those living with DMD and the research teams working to find new treatments.

About Vamorolone

Vamorolone is an exciting new investigational drug candidate that offers a unique mode of action, binding to the same receptor as glucocorticoids but with modified downstream activity. This could potentially give it the ability to provide efficacy without the safety concerns of existing steroids, making it a promising alternative for the treatment of DMD in children and adolescents.

Results from the pivotal VISION-DMD study showed that vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and had a good safety and tolerability profile, with the most commonly reported adverse events being cushingoid features, vomiting and vitamin D deficiency.

Although vamorolone is not yet approved for use, the findings from the VISION-DMD study give reason for optimism that it could become a valuable treatment option in the future.

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