FDA Grants Priority Review for CUTX-101

Sentynl Therapeutics, a subsidiary of Zydus Lifesciences, has received Priority Review from the U.S. FDA for its New Drug Application (NDA) for CUTX-101, aimed at treating Menkes disease, a rare and fatal pediatric disorder linked to mutations in the ATP7A gene. The FDA has set a target action date for June 30, 2025. Clinical trials indicate that early treatment with CUTX-101 results in a nearly 80% reduction in mortality risk, with median survival improving significantly from 16.1 to 177.1 months compared to untreated controls. Menkes disease affects copper transport in the body, leading to severe health issues and often resulting in death by age three without treatment. The drug has also received multiple designations, including Breakthrough Therapy and Orphan Drug status, reflecting its potential impact on a community with significant unmet needs. This milestone marks a critical step toward potentially providing the first FDA-approved treatment for Menkes disease.