FDA approval of Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy.- Santhera Pharma

“This new drug approval gives Duchenne patients and their families one more reason for hope,” said Pat Furlong, Founding President & CEO Parent Project Muscular Dystrophy (PPMD). “Steroids are considered standard of care for Duchenne, due to their valuable role in slowing disease progression. However, they often come with considerable side effects. Vamorolone has the potential to be an alternative steroid with a better tolerability profile addressing an important unmet medical need for patients.”

The FDA approval of Agamree was based on the data from the pivotal Phase IIb VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, Agamree was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. 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 behavior. The studies in the development program were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.