Monday, July 15, 2024

Santhera’s Vamorolone Nears Regulatory Approval for Duchenne Muscular Dystrophy Treatment

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Swiss biotech company Santhera is on the verge of achieving its first regulatory approval for the Duchenne muscular dystrophy (DMD) treatment, vamorolone, following a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

The CHMP has endorsed the approval of the drug, marketed under the trade name “Agamree,” for the treatment of DMD patients aged four years and older. Pending full approval in the European Union, typically following a CHMP recommendation by a few weeks, Santhera plans to launch Agamree in Germany in the first quarter of the upcoming year.

Santhera anticipates that Agamree could become the first drug to receive full approval from the European Medicines Agency for DMD treatment. Additionally, vamorolone has been submitted for approval in the United States, with a decision expected later this month from the U.S. Food and Drug Administration (FDA).

This recommendation from the CHMP marks a significant turnaround for Santhera, which previously had to undergo staff reductions and operational restructuring after its initial DMD candidate, idebenone, failed a phase 3 trial in 2020. Vamorolone was licensed by U.S. biotech company ReveraGen BioPharma in 2020, shortly after Santhera ceased the development of idebenone for DMD.

Santhera’s CEO, Dario Eklund, expressed excitement about the CHMP’s positive opinion, emphasizing the critical medical need for an effective and well-tolerated treatment for DMD. He also noted that Santhera is prepared to make Agamree available to EU patients as soon as marketing authorization is granted by the European Commission.

The CHMP’s recommendation is primarily based on data from the phase 2b VISION-DMD study, which demonstrated that vamorolone had positive effects on mobility measures, such as the six-minute walk test (6MWT) and time to stand velocity (TTSTAND), compared to a placebo over 24 weeks, with these benefits sustained at 48 weeks in DMD patients aged four to seven.

Furthermore, boys treated with vamorolone maintained growth similar to those receiving a placebo, while those treated with the standard DMD therapy, corticosteroid prednisone, experienced growth stunting. Patients who switched from prednisone to vamorolone after 24 weeks were able to resume height growth over the remainder of the study.

Trials of vamorolone are also ongoing in younger DMD patients aged two to four and those aged seven to 18, as well as in patients with Becker muscular dystrophy (BMD), a less common form of muscular dystrophy with milder symptoms.

Should vamorolone gain FDA approval, it will be commercialized in the United States by Catalyst Pharmaceuticals, which secured licensing rights to the drug for $90 million earlier this year. Santhera has previously estimated that the drug could generate annual sales exceeding $500 million in both the U.S. and Europe if approved.

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