Business Wire·Sep 25, 2026, 7:00 PM ET

Mirum Pharmaceuticals and Incyte Announce U.S. FDA Approval of Atebrioz™ (zilurgisertib) for Adult and Pediatric Patients with Fibrodysplasia Ossificans Progressiva

MIRM INCY

FDA approved Atebrioz (zilurgisertib) as a once-daily oral ALK2 inhibitor for patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP) to reduce total new heterotopic ossification volume
Recommended dosage is 100 mg taken orally once daily
Atebrioz is expected to be commercially available in the United States in October through the Mirum Access Plus program, with eligible patients paying as little as $0 per month
Clinical efficacy was demonstrated in Cohort 1 of the PROGRESS study, where patients treated with zilurgisertib showed a mean decrease of 3.2 cm³ in new HO lesion volume compared to a 24.6 cm³ increase in the placebo group over 24 weeks
Treatment effects were maintained through Week 48 in the open-label extension phase of the study
Zilurgisertib was generally well tolerated, with common adverse reactions including headache, arthralgia, upper respiratory tract infection, epistaxis, and nausea
No adverse events led to treatment discontinuation or dose reduction during the placebo-controlled period
Incyte received a Rare Pediatric Disease Priority Review Voucher from the FDA for this approval
A marketing authorization application for the European Union is currently under review by the EMA
The PROGRESS pediatric development program continues enrollment in Cohort 3 for children aged 2 to <12 years following completion of Cohort 2 enrollment for those aged 6 to <12 years
Atebrioz carries warnings regarding fetal harm, requiring effective contraception for patients of reproductive potential
Zilurgisertib was developed by Incyte and licensed to Mirum Pharmaceuticals for worldwide development and commercialization