
Fibrodysplasia ossificans progressiva (FOP) is an ultra‐rare genetic disorder characterized by progressive heterotopic ossification (HO), often heralded by flare‐ups, leading to reduced movement and life expectancy. This placebo‐controlled, double‐blind trial (NCT02190747) evaluated palovarotene, an orally bioavailable selective retinoic acid receptor gamma agonist, to prevent HO in patients with FOP. At Week 12, the proportions were 95.0% with palovarotene 10/5 mg; 88.9% with palovarotene 5/2.5 mg; 77.8% with placebo (Cochran‐Armitage trend test: p=0.15). Although these findings were not statistically significant, they support further evaluation of palovarotene for preventing HO in FOP in larger studies.
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