FDA Approves Third FOP Treatment, Atebrioz

The FDA has approved Atebrioz (zilurgisertib) tablets for adults and children 12 and older with fibrodysplasia ossificans progressiva, according to the FDA's announcement. This is the third approved treatment for the rare genetic disease, which is caused by a mutation in a bone growth receptor. In FOP, connective tissue such as muscle and tendon gradually turns to bone, leading to progressive loss of movement and early death.

The approval rests on a randomized, double blind, placebo controlled trial of 63 patients who took Atebrioz or placebo once daily for 24 weeks, followed by a long open label extension. Using whole body CT scans, researchers measured the change in volume of new heterotopic ossification. Patients on Atebrioz saw an average 3.2 cubic centimeter decrease, while the placebo group had a 24.6 cubic centimeter increase, a meaningful difference in a disease with no natural mechanism to reverse this process.

The recommended starting dose is 100 mg orally once daily. The FDA warns Atebrioz can cause fetal harm based on animal data, so patients of reproductive potential are advised to use effective contraception. Common side effects reported include headache, joint pain, upper respiratory infection, nosebleeds, and nausea. The drug received fast track, priority review, and orphan drug designation.

This article is for education only. It is not medical advice. Compounds discussed here are sold for research purposes. Talk to a licensed clinician before making health decisions.