FDA approves Agamree for Duchenne muscular dystrophy

The FDA has approved a 40 mg/mL oral suspension of Agamree to treat Duchenne muscular dystrophy in patients aged 2 years and older, according to a release from the manufacturer.

Approval was based on data from the phase 2b VISION-DMD study, supplemented with safety information from three additional open-label studies, Santhera Pharmaceuticals said in the release.

The trials, which were conducted by Santhera partner ReveraGen BioPharma at 32 centers across 11 countries, saw Agamree (vamorolone) administered at doses ranging from 2 mg/kg to 6 mg/kg per day for up to 48 months. Data show the novel corticosteroid demonstrated greater efficacy and reduction of adverse events relating to bone health, growth and behavior compared with the current standard of care, the company said.

“This is a hugely important moment for [Duchenne muscular dystrophy] patients who need an efficacious and well-tolerated therapy for this debilitating condition,” Santhera CEO Dario Eklund said in the release.

Santhera said it expects to transfer U.S. marketing authorization for the therapy to its partner, Catalyst Pharmaceuticals, which holds an exclusive license for marketing in North America and plans to launch the product in the first quarter of 2024.

“Today’s news represents the culmination of many years of research to bring vamorolone to patients,” Eric Hoffman, PhD, president and CEO of ReveraGen BioPharma, said in the release. “We are pleased to have worked alongside Santhera, the [Duchenne muscular dystrophy] patient community, researchers and health care practitioners to reach this important milestone.”