FDA Approves Regeneron Pasatru for FOP

Regeneron’s garetosmab-grts, sold as Pasatru, received FDA approval as the second treatment for fibrodysplasia ossificans progressiva (FOP), a ultra-rare condition where soft tissues progressively turn into bone. The approval follows the OPTIMA trial, which showed Pasatru reduced new bone lesions by about 90% over 56 weeks and cut clinician‑assessed flare-ups by a large margin, potentially outperforming the first approved therapy, Sohonos. Pasatru’s mechanism targets activin A, a key driver in FOP, and is administered intravenously every four weeks, with dose options of 10 mg/kg or 3 mg/kg if tolerated. The drug’s development is spurring continued regulatory review in Europe and Japan, while Regeneron has not disclosed a U.S. list price; Sohonos previously carried a price around $624,000 per year. FOP affects roughly 900 people worldwide, with many patients wheelchair-bound by age 30 and a median life expectancy around 56, highlighting Pasatru’s potential impact on mobility and quality of life for adults with FOP. The FDA approval marks a milestone for Regeneron and the FOP community, offering a second, clinically meaningful option for managing the disease’s progression and pain.