FDA Accepts Regeneron (REGN) BLA for Garetosmab Under Priority Review

Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) is one of the best value stocks to buy now. On February 19, Regeneron Pharmaceuticals announced that the FDA accepted its BLA for garetosmab for Priority Review. This investigational monoclonal antibody is designed to treat adults with fibrodysplasia ossificans progressiva/FOP, which is an ultra-rare and devastating genetic disorder where muscles and connective tissues are progressively replaced by bone.

If approved, garetosmab would be the first treatment proven to significantly reduce both the number and volume of these abnormal bone lesions, with an FDA decision expected by August this year. The application is supported by the Phase 3 OPTIMA trial, which showed dramatic results in adult patients. Over 56 weeks, patients receiving garetosmab experienced a 90% to 94% reduction in the total number of new bone lesions compared to those on a placebo. Furthermore, a post-hoc analysis revealed a more than 99% reduction in the mean total volume of new abnormal bone formation.

The treatment works by neutralizing Activin A, which is a protein that Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) scientists identified as a critical driver of the locking bone growth characteristic of FOP. FOP is a relentless condition that often leads to a complete loss of mobility; most patients require a wheelchair by age 30, and the median survival age is ~56 years. Regeneron plans to initiate the OPTIMA 2 trial later this year to evaluate the safety and efficacy of garetosmab in children and adolescents.

Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) discovers, invents, develops, manufactures, and commercializes medicines to treat various diseases worldwide.

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