Home / Health / FDA Greenlights Atebrioz for Rare FOP Condition
FDA Greenlights Atebrioz for Rare FOP Condition
26 Sep
Summary
- FDA approved Atebrioz to reduce new heterotopic ossification in FOP.
- Atebrioz is a once-daily oral ALK2 inhibitor targeting the disease pathway.
- Patient support program MAP offers financial assistance and access support.
In a significant development for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved Atebrioz™ (zilurgisertib) tablets. This oral medication is now available to reduce the volume of new heterotopic ossification (HO) in adult and pediatric patients aged 12 and older diagnosed with fibrodysplasia ossificans progressiva (FOP). Atebrioz targets the abnormal activation of ALK2, a pathway central to FOP's progression.
The approval, announced by Mirum Pharmaceuticals and Incyte, brings a much-needed new therapeutic option for individuals living with this debilitating condition. Atebrioz is a once-daily oral treatment, and its efficacy was demonstrated in the PROGRESS study. Patient support will be provided through the Mirum Access Plus (MAP) program, assisting with insurance, access, and financial aid.
Commercial availability of Atebrioz is anticipated in the U.S. in October. The FDA's decision was accompanied by a Rare Pediatric Disease Priority Review Voucher for Incyte. Meanwhile, a marketing authorization application for zilurgisertib is under review by the European Medicines Agency (EMA), with ongoing clinical trials evaluating the drug in younger pediatric FOP patients.
