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Garetosmab Biologics License Application Accepted for FDA Priority Review for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
FOP is an ultra-rare genetic disorder characterized by abnormal bone formation that infiltrates muscles, tendons, ligaments and other connective tissues,
About this update from Regeneron Pharmaceuticals, Inc.
[{"type":"text","content":"FOP is an ultra-rare genetic disorder characterized by abnormal bone formation that infiltrates muscles, tendons, ligaments and other connective tissues, resulting in significant disability If approved, garetosmab would be the first and only available treatment shown to reduce the number and volume of new heterotopic bone lesions in adults with FOP TARRYTOWN, N.Y., Feb. 19, 2026 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for garetosmab for the treatment of adults with fibrodysplasia ossificans progressiva (FOP). Garetosmab is a monoclonal antibody that blocks Activin A, a protein that Regeneron scientists discovered to be critical in the development of heterotopic ossification (HO) lesions in people with FOP. The target action date for the FDA decision is August 2026. FOP is a relentless, ultra-rare genetic disorder in which muscles, tendons, ligaments and other connective tissues are progressively infiltrated by abnormal bone formation, a process known as HO, which results in significant disfunction of these structures and skeletal deformity. HO of the jaw, spine, hip and rib cage can make it difficult to speak, eat, walk or breathe, leading to weight loss and escalating loss of mobility. Most people with FOP are wheelchair bound by 30 years old, and the median age of survival is approximately 56 years. Approximately 900 people are diagnosed with FOP worldwide, with many others thought to remain undiagnosed or misdiagnosed. The BLA is supported by efficacy and safety data from the positive Phase 3 OPTIMA trial evaluating garetosmab in adults with FOP. Both garetosmab doses (3 mg/kg and 10 mg/kg) evaluated in the trial were highly efficacious in reducing the total number and volume of new HO lesions at 56 weeks, compared to placebo. Regarding the primary endpoint analysis of reduction in total number of new HO lesions compared to placebo (n=21), those receiving the 3 mg/kg dose (n=19) experienced a 94% reduction (1 lesion vs. 19 lesions; p=0.0274), while those receiving the 10 mg/kg dose (n=23) experienced a 90% reduction (2 lesions vs. 19 lesions; p=0.0260). A post-hoc analysis also found both doses of garetosmab demonstrated a greater than 99% reduction in mean total ...