Incyte and Mirum Pharmaceuticals announced positive results from a pivotal Phase 2 study of zilurgisertib. The drug targets Fibrodysplasia Ossificans Progressiva (FOP), a rare disease where bone forms in connective tissues. Data presented at the ENDO 2026 conference showed zilurgisertib reduced new bone lesion formation by 81%. This efficacy was measured against a placebo over a 24-week period. The volume of these lesions decreased by 99.9%.
Clinical benefits were sustained through 48 weeks of treatment. No new lesions were observed in patients during the subsequent open-label extension period.
The FDA accepted the New Drug Application for zilurgisertib and granted it Priority Review. This designation shortens the regulatory review timeline for the treatment. The agency set a PDUFA target action date of September 26, 2026.