FDA Approves New Duchenne Muscular Dystrophy Drug

The FDA has approved Agamree (vamorolone) to treat patients over the age of 2 with Duchenne muscular dystrophy. Duchenne is a rare genetic disease that is characterized by a mutation in the dystrophin gene. It causes the muscles in the body to become weak and damaged over time and is eventually fatal. Duchenne affects about 1 in 3,500 to 5,000 males born worldwide.

Agamree, which was developed by Santhera, is a novel corticosteroid treatment option. In July 2023, Catalyst Pharmaceuticals secured the exclusive North American license and commercial rights for Agamree, and Catalyst plans to launch in the first quarter of 2024. Pricing information will be available at that time, according to a spokesperson.

“Steroids are considered standard of care for Duchenne, due to their valuable role in slowing disease progression,” Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD), said in a press release. “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’s approval was based on the data from the pivotal phase 2b VISION-DMD, study along with safety information collected from three open-label studies. Compared with current standard of care corticosteroids, Agamree exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior.

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.