Regeneron announced that the U.S. Food and Drug Administration has accepted its Biologics License Application for garetosmab with priority review for the treatment of adults with fibrodysplasia ossificans progressiva (FOP). The acceptance, made on February 19 2026, grants the company an accelerated review timeline and a target decision date of August 2026, potentially bringing the first disease‑modifying therapy for this ultra‑rare disorder to market.
The Phase 3 OPTIMA trial demonstrated that both 3 mg/kg and 10 mg/kg doses of garetosmab reduced the number of new heterotopic bone lesions by 94 % and 90 % respectively, compared with placebo over 56 weeks. A post‑hoc analysis showed a greater than 99 % reduction in the volume of new lesions for both doses. Safety data were manageable, with serious treatment‑emergent adverse events occurring in one patient on the 3 mg/kg dose, two patients on the 10 mg/kg dose, and two patients on placebo. Common reactions included epistaxis, increased hair growth, abscess, and acne.
FOP affects roughly 900 patients worldwide, making it one of the rarest genetic disorders. Garetosmab’s approval would establish Regeneron as a leader in a niche market with no approved disease‑modifying therapies, offering a significant revenue opportunity and reinforcing the company’s rare‑disease portfolio. The company plans to initiate a Phase 3 study in adolescents and children later in 2026, expanding the potential patient base.
Regeneron’s leadership has emphasized its commitment to rare diseases. George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer, noted that the OPTIMA trial success “is a direct result of Regeneron’s relentless pursuit of science and use of proprietary technologies to improve the lives of people with debilitating and life‑threatening diseases, no matter their prevalence.” The acceptance also follows prior Fast Track and orphan designations in the U.S. and EU, underscoring the company’s strategic focus on translating foundational science into therapies for underserved populations.
The priority review designation places garetosmab ahead of the typical 10‑month review window, potentially shortening the path to approval by several months. With a target decision date in August 2026, Regeneron could bring a first‑in‑class treatment to the approximately 900 patients worldwide, aligning with its broader goal of closing the gap in FDA‑approved rare‑disease therapies. The milestone also signals confidence from the FDA in the drug’s safety and efficacy profile, bolstering Regeneron’s reputation as a leader in rare‑disease innovation.
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