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News
The FDA has approved zilurgisertib (Atebrioz) tablets for patients aged 12 and older with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition that causes muscles and soft tissues to turn into bone. It's now the third approved therapy for FOP, backed by trial data showing a meaningful reduction in abnormal bone growth compared to placebo.
The FDA has approved zilurgisertib (Atebrioz, Mirum Pharmaceuticals) tablets for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare and debilitating genetic disease in which soft tissues progressively turn into bone. The approval covers patients aged 12 and older and makes Atebrioz the third available treatment for FOP, joining Garetosmab-grts (Pasatru) and palovarotene (Sohonos).
The green light was supported by a randomized, double-blind, placebo-controlled trial in 63 FOP patients, followed by a lengthy open-label extension. Whole-body CT scans tracked changes in heterotopic ossification — the hallmark of FOP — over the study period.
By the Numbers:
Why it matters: FOP is an extraordinarily rare and progressive condition with no cure, making every new approved therapy a significant milestone for patients. Having three treatment options now available expands the clinical toolkit and offers hope for better disease management in this vulnerable population.
