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Catalyst Pharmaceuticals: FDA Approval of AGAMREE® (vamorolone)

October 27, 2023

Defeat Duchenne Canada is excited to share that Catalyst Pharmaceuticals, Inc. has reported that Santhera Pharmaceuticals (“Santhera”) has obtained U.S. Food and Drug Administration (“FDA”) approval for AGAMREE® (vamorolone), a dissociative steroid indicated for individuals diagnosed with Duchenne muscular dystrophy from age two years and older.

AGAMREE offers a novel corticosteroid treatment option for DMD, addressing a significant unmet medical need. Compared with current standard-of-care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory, and behaviour.

Defeat Duchenne Canada is proud to have invested $750,000 to fund Canadian clinical trial research for 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 expected on the EMA drug label in Europe (4 years and older).

We are grateful to Drs. Jean Mah (Calgary), Kathy Selby (Vancouver), Hugh McMillan and Hanns Lochmuller (Ottawa), and Hernan Gonorazky (Toronto) and their teams, as well as the Canadian families participating in the VBP15-006 that made this broad age label possible.

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

“We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with Duchenne Muscular Dystrophy and potentially other chronic inflammatory diseases. The approval of AGAMREE underscores the potential of reshaping the DMD treatment paradigm for this life-threatening rare disease.

Our unwavering commitment extends beyond this important milestone as we are resolute in our mission to ensure that DMD patients in the U.S. have access to this treatment option as we believe that AGAMREE may offer the potential of increasing the duration of ambulation and mobility in these patients, thereby significantly improving their overall quality of life. We expect to launch the product in the first quarter of 2024”.

Patrick J. McEnany, Chairman and CEO of Catalyst Pharmaceuticals

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

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