October 13, 2023 / Clinical Trials,Research

Santhera Receives Positive European Medicines Agency (EMA) CHMP Opinion Recommending Approval of Vamorolone in European Union

Santhera Pharmaceuticals has announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion in favor of approval of vamorolone (AGAMREE®) for the treatment of Duchenne muscular dystrophy patients aged 4 years and older. Vamorolone is an investigational dissociative steroid for individuals with Duchenne.

The European Medicines Agency (EMA) is the European Union’s equivalent to the U.S. Food and Drug Administration (FDA). A marketing authorization decision from the European Commission is expected within approximately two months of the positive CHMP opinion. 

Additionally, a new drug application (NDA) for vamorolone in Duchenne is under review in the U.S. by the FDA with a Prescription Drug User Fee Act (PDUFA) date for its regulatory decision on approval set for October 26, 2023. While the CHMP’s opinion does not directly impact the FDA’s consideration of vamorolone’s NDA, this positive news is promising for the Duchenne community in the U.S. 

In Santhera’s pivotal VISION-DMD study, patients treated with vamorolone on average maintained growth similar to those treated with placebo, while those treated with prednisone on average experienced growth stunting. Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study. In its recommendation for approval, the CHMP acknowledged the positive benefit-risk profile of vamorolone in this patient population, including certain safety benefits of vamorolone compared to standard of care corticosteroids in the treatment of Duchenne.

PPMD is optimistic about another potential therapeutic option for Duchenne and we are proud to have collaborated with other Duchenne groups to support the early development of vamorolone.

Read Santhera’s press release here.

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