FDA approves Atebrioz for FOP patients aged 12 and older

The FDA approved Atebrioz (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification, or bone formation outside the skeleton, in adults and pediatric patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP). The FDA described FOP as a rare genetic disease. Healio reported that Atebrioz is the third approved treatment for the condition.

The approval was based on a randomized, double-blind, placebo-controlled trial in which 63 patients received Atebrioz 100 mg or placebo once daily for 24 weeks. At Week 24, patients receiving Atebrioz had an average 3.2 cm³ decrease in the volume of total new heterotopic ossification, while the placebo group had a 24.6 cm³ increase. The trial was followed by a 292-week, single-arm, open-label extension period.

The FDA said the most common side effects are headache, joint pain, upper respiratory tract infection, nosebleeds and nausea. It also said Atebrioz can cause fetal harm based on animal studies, and patients of reproductive potential should use effective contraception.