Zydus Lifesciences, through its U.S.-based subsidiary Sentynl Therapeutics, has achieved a critical milestone with the U.S. FDA granting priority review to its New Drug Application (NDA) for CUTX-101 (Copper Histidinate). The product, aimed at treating Menkes disease, a rare and fatal pediatric disorder, could become the first FDA-approved therapy for this condition.
Key Highlights:
- Priority Review: CUTX-101 has been granted a six-month priority review with a target action date of June 30, 2025.
- Breakthrough Designations: The drug has earned multiple designations, including FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug status.
- Clinical Results: Early treatment with CUTX-101 demonstrated an 80% reduction in mortality risk compared to untreated historical controls, with median survival improving to 177.1 months from 16.1 months.
Menkes disease, caused by ATP7A gene mutations, affects copper transport in the body, leading to severe neurological and physical symptoms. Without treatment, life expectancy is often limited to 2–3 years.
