FDA approves Atebrioz for rare bone disorder FOP

The U.S. Food and Drug Administration has approved Atebrioz (zilurgisertib) for the treatment of adults and pediatric patients aged 12 and older with fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare genetic disorder characterized by the progressive formation of bone within muscles, tendons, and ligaments.

Developed by Incyte Corporation and licensed to Mirum Pharmaceuticals for worldwide commercialization, Atebrioz is an oral, once-daily tablet. Clinical data from a 24-week study showed that patients treated with the drug experienced a reduction in the mean volume of new bone lesions compared to those receiving a placebo.

Mirum Pharmaceuticals expects to launch the treatment in the United States in October. The company plans to facilitate access through its Mirum Access Plus (MAP) patient support program, which provides financial assistance and insurance coverage support. While approved in the U.S., regulatory reviews for the drug are ongoing in other regions, including a marketing authorization application submitted to the European Medicines Agency.