June 14, 2026
3 min read
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Garetosmab reduces lesion formation, volume in patients with rare bone disease
CHICAGO — Garetosmab reduced new heterotopic bone lesions in patients with fibrodysplasia ossificans progressiva at 56 weeks compared with placebo, according to results of the OPTIMA trial presented at ENDO 2026. “[Fibrodysplasia ossificans progressiva] is an ultrarare disorder that affects about one in 1 million of the population,” Richard Keen, MD, PhD, director of the Centre
TL;DRAI
Regeneron's garetosmab reduced new heterotopic bone lesions by 94-99% versus placebo in fibrodysplasia ossificans progressiva patients (1-2 vs. 5 cases, 56-week OPTIMA phase 3 trial). Well-tolerated activin A inhibitor establishing clear path toward first approved treatment for ultrarare genetic disorder.
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