Zydus Lifesciences Ltd Receives USFDA Complete Response Letter for CUTX-101 NDA, Eyes Next Steps for Menkes Disease Treatment

Zydus Lifesciences Ltd, through its wholly-owned subsidiary Sentynl Therapeutics, has informed the receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration (USFDA) regarding its New Drug Application (NDA) for CUTX-101 (Copper Histidinate), a potential treatment for the rare pediatric condition Menkes disease. The CRL highlights areas needing further clarification but confirms no concerns with drug efficacy or safety.

 

Key Highlights of the USFDA Communication

 

The CRL follows a thorough review process including a CGMP inspection at the manufacturing facility for CUTX-101.

 

Zydus and Sentynl recently responded to USFDA’s September 2025 re-inspection findings, demonstrating compliance with CGMP standards and are now awaiting the Establishment Inspection Report (EIR).

 

USFDA did not flag any issues regarding the effectiveness or safety data underpinning CUTX-101’s clinical profile, underscoring the drug's therapeutic potential.

 

The company plans to meet with USFDA to discuss the CRL contents and outline the path forward for NDA resubmission.

 

Strategic Importance of CUTX-101

Menkes disease is a rare, often fatal genetic disorder caused by copper transport deficiencies. CUTX-101 has shown promising clinical efficacy, reducing mortality risk by nearly 80% with early treatment, positioning it as a breakthrough therapy in this unmet medical area.

 

Outlook and Next Steps

Zydus Lifesciences is committed to advancing CUTX-101 through the regulatory process and delivering a transformative therapy for patients affected by Menkes disease. The company’s engagement with USFDA reflects proactive efforts to address regulatory feedback and expedite product availability.

 

Sources: PR Newswire, CNBC TV18, Zydus Lifesciences official statements, Sentynl Therapeutics press releases.