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Santhera Pharmaceuticals: AGAMREE® (Vamorolone) Approved in the European Union

December 19, 2023

Santhera Pharmaceuticals has announced that AGAMREE® (vamorolone) has been approved in the European Union (EU) for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older, independent of the underlying mutation and ambulatory status.

  • European Commission (EC) grants marketing authorization for AGAMREE® for the treatment of Duchenne muscular dystrophy (DMD) in patients aged four years and older.
  •  AGAMREE® is the only approved medication in the European Union (EU) for treating DMD and the first DMD treatment approved in both the U.S. and EU
  • First commercial launch of AGAMREE®, in Germany, expected in Q1-2024
  • EMA acknowledges the safety benefits of AGAMREE® with regards to preserving bone health and maintaining growth compared to standard-of-care corticosteroids

The European Medicines Agency (EMA) acknowledged the clinically important safety benefits of AGAMREE with regard to maintaining normal bone metabolism, density, and growth compared to standard-of-care corticosteroids, alongside similar efficacy. In addition, patients who switched from a standard-of-care corticosteroid to AGAMREE maintained the efficacy benefit while recovering their growth and bone health.

 “We are thrilled to receive approval from the EC to bring AGAMREE to patients in the EU, which highlights the favorable safety and tolerability profile over conventional corticosteroids, including benefits for bone health and growth.”

Dario Eklund, CEO of Santhera

“The World Duchenne Organization is happy to see the development of AGAMREE, during which the involvement of many Duchenne patient organizations has been instrumental by providing early funding and contributing to clinical trial design, outcomes and recruitment, led to the first full market authorization in the European Union for treating Duchenne muscular dystrophy.”

Elizabeth Vroom, Chair of the World Duchenne Organization

Defeat Duchenne Canada is proud to have invested $750,000 to fund Canadian clinical trial research for vamorolone in boys 2-4 and 7-18 at five expert academic medical centers in Canada, enabling approval for expanded age access worldwide.

“Defeat Duchenne Canada was the key supporter of the Canada-only VBP15-006 trial that studied vamorolone in 2 to 4-year-old and 7 to 18-year-old DMD boys. The VBP15-006 clinical trial provided vital data to enable the broad age range on the FDA drug label (2 years and older) and now the approval by the EMA in Europe (4 years and older).”

Dr. Eric Hoffman, Professor of Pharmaceutical Sciences at Binghamton University – State University of New York

The marketing authorization will be valid in all 27 member states of the EU as well as Iceland, Liechtenstein and Norway, with Santhera expecting to begin a commercial launch in Germany in Q1-2024. Santhera announced on October 27, 2023, that the FDA approved AGAMREE oral suspension 40 mg/ml in the United States for the treatment of DMD in patients two years of age and older. Catalyst Pharmaceuticals holds an exclusive license for AGAMREE in North America and plans to launch the product in the U.S. in Q1-2024.

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