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Santhera Pharmaceuticals: European Medicines Agency Validates Marketing Authorization Application for Vamorolone

October 13, 2023

Santhera Pharmaceuticals has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending the approval of AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy. Highlights include:

  • The Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in children and adults aged four years and older.
  • European Commission (EC) decision on marketing authorization is expected in late 2023.
  • AGAMREE (vamorolone) could become the first drug fully approved by the European Medicines Agency (EMA) to treat patients with DMD.
  • Anticipation of vamorolone approval in the U.S. on the PDUFA date of October 26, 2023. Subject to approval, Santhera’s licensing partner, Catalyst Pharmaceuticals, plans to launch vamorolone in the U.S. in Q1-2024.

In its recommendation for approval, the CHMP acknowledges the positive benefit-risk profile of AGAMREE (vamorolone) in this patient population, including certain safety benefits of AGAMREE (vamorolone) compared to standard-of-care corticosteroids in the treatment of Duchenne. Santhera plans for a first commercial launch in Germany in Q1-2024, subject to approval by the EC.

“We are thrilled about the CHMP’s positive opinion, which recognizes the urgent medical need for an effective and well tolerated treatment for this devastating disease. We can now execute on our plans to ensure AGAMREE is made available to patients in the EU as soon as the European Commission marketing authorization is received. The recommendation by the CHMP is a testament to the dedication and collaboration of all those involved in the development of vamorolone, including ReveraGen Biopharma, the DMD patient community, researchers, healthcare professionals, and our employees.”

Dario Eklund, CEO of Santhera

The clinical evidence for the efficacy and safety of AGAMREE (vamorolone) in the regulatory submission was derived from the positive pivotal VISION-DMD study and three open-label studies (including extensions) in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months, as well as an external comparator study and several clinical pharmacology studies. The studies were carried out by Santhera’s partner, ReveraGen, and 32 academic clinical trial centers in 11 countries.

Defeat Duchenne Canada invested $750,000 to fund the Canadian clinical trial research for boys 7-18, which has enabled approval for expanded age access worldwide.

The support from Defeat Duchenne Canada was critical for this program, as it enabled ReveraGen to carry out a clinical trial in a broad age range of DMD boys, including 2-4 years and 7-18 years old, at five expert academic medical centers in Canada.

The data from this Canada-only age expansion trial provided key data to increase the age range of boys treated with vamorolone from our previous clinical trials (narrow 4 to <7 years age range), to the much broader 2 to 18 years, inclusive of older boys transitioned from prednisone or deflazacort, to vamorolone – the only experience worldwide. The Canada-only VBP15-006 clinical trial supported by Defeat Duchenne Canada is being carried out at Calgary (Dr. Jean Mah), Vancouver (Dr. Kathy Selby), Ottawa (Dr. Hanns Lochmuller), Toronto (Dr. Hernan Gonorszky).

Dr. Eric Hoffman, CEO ReveraGen and Professor Pharmaceutical Sciences, Binghamton University – State University of New York

In the pivotal VISION-DMD study, boys treated with vamorolone, on average, maintained growth similar to those treated with a placebo, while those treated with prednisone 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.

Available data show that vamorolone, unlike corticosteroids, has no restriction of growth and no negative effects on bone metabolism, as demonstrated by the normal bone formation and bone resorption serum markers.

Santhera will continue to collect data to further characterize the long-term effectiveness and safety differentiation of vamorolone.

A marketing authorization decision from the EC is expected within approximately two months of the positive CHMP opinion. Subject to approval, AGAMREE (vamorolone) will be the first and only medicinal product fully approved by the EMA for the treatment of Duchenne, and the marketing authorization will be valid in all 27 member states of the European Union as well as Iceland, Liechtenstein and Norway. Potential launches of AGAMREE (vamorlone) in the EU, with Germany taking the lead, are planned to start in Q1-2024.

In the U.S., the FDA established the target Prescription Drug User Fee Act (PDUFA) action date for its regulatory decision on the vamorolone NDA as October 26, 2023. Subject to approval, Santhera’s licensing partner, Catalyst Pharmaceuticals, plans to launch vamorolone in the U.S. in Q1-2024.

Defeat Duchenne Canada will keep the Duchenne community updated on pending approvals and provide more information on plans for Canada as they become available.

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