New Delhi: Ahmedabad-based Zydus Lifesciences on Monday said its US subsidiary Sentynl Therapeutics has received acceptance from the US Food and Drug Administration (FDA) for the resubmission of its New Drug Application (NDA) for CUTX-101, an investigational therapy for Menkes disease, a rare pediatric genetic disorder.
The FDA has classified the resubmission as a Class I response, assigning a new Prescription Drug User Fee Act (PDUFA) target action date of January 14, 2026, Sentynl said in a statement.
The NDA resubmission follows a Complete Response Letter (CRL) issued by the regulator in September 2025, which flagged observations related to current good manufacturing practice (cGMP) compliance at the manufacturing site. The company clarified that the CRL did not raise any concerns on the efficacy or safety data of the drug candidate.
CUTX-101 (copper histidinate) is being developed for the treatment of Menkes disease, an X-linked recessive disorder affecting copper metabolism, primarily in male infants. If approved, it would be the first FDA-approved treatment for the condition.
According to the company, clinical data from NIH-led studies showed statistically significant improvement in overall survival, with early-treated patients demonstrating nearly an 80% reduction in mortality risk compared to untreated historical cohorts.
CUTX-101 has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations from the FDA, while the European Medicines Agency has also granted it orphan status.
Zydus Lifesciences said the development reinforces its focus on rare and orphan diseases through its US operations, as it continues to expand its specialty and innovation-led portfolio.