Pediatric Drug CUTX-101 from Zydus Lifesciences Flagged by USFDA

Zydus Lifesciences arm’s pediatric Menkes disease drug, CUTX-101, has been flagged by the US Food and Drug Administration (USFDA) through a Complete Response Letter (CRL). The regulatory notice relates to findings from a current Good Manufacturing Practices (CGMP) inspection at the facility where CUTX-101 is produced. 

The USFDA outlined certain requirements that must be addressed before moving forward with the approval process.

Company Response and Ongoing Regulatory Process

Zydus Lifesciences noted that it submitted responses to the USFDA following a re-inspection in September 2025, confirming the facility’s CGMP compliance. The company is currently awaiting the Establishment Inspection Report (EIR). 

Sentynl Therapeutics, the US subsidiary handling CUTX-101, plans to request a meeting with the USFDA to clarify the CRL and discuss resubmission of the NDA. Importantly, the CRL did not raise concerns regarding the drug’s safety or efficacy, indicating that CUTX-101 remains viable for approval once regulatory feedback is addressed.

Understanding CUTX-101 and Menkes Disease

Menkes disease is a rare X-linked recessive pediatric genetic disorder that affects copper absorption and transport. The condition is estimated to occur in 1 in 34,810 to 1 in 8,664 live male births. CUTX-101, a subcutaneous injectable formulation of copper histidinate, helps restore copper levels in patients, ensuring proper copper homeostasis. 

The drug has demonstrated significant improvement in overall survival for early-treated patients, supporting its clinical potential. CUTX-101 was previously granted Priority Review status by the USFDA.

Zydus Lifesciences Share Price and Market Impact

Following the announcement of the CRL, Zydus Lifesciences share price showed resilience. Shares of the company closed at ₹993.90, up ₹12.20 or 1.24%, on the BSE. The positive market reaction reflects investor confidence in the company’s ability to address the USFDA’s observations and the underlying potential of CUTX-101 in treating Menkes disease.

Read more:Tata Investment Corporation Share Price Hits 52-Week High

Future Steps and Company Commitment

Zydus Lifesciences arm remains committed to progressing CUTX-101 despite the regulatory setback. The company emphasises that the CRL does not indicate any concerns with the drug’s efficacy or safety. 

By engaging with the USFDA and addressing the outlined points, Zydus aims to resubmit the NDA promptly, continuing its efforts to provide innovative treatment options for pediatric patients with Menkes disease.

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