Sentynl Therapeutics Responds to FDA's Complete Response Letter on CUTX-101 Approval for Menkes Disease

Sentynl Therapeutics Receives Complete Response Letter for CUTX-101

Sentynl Therapeutics, Inc., a California-based biopharmaceutical firm and subsidiary of Zydus Lifesciences, has recently been in the spotlight following the U.S. Food and Drug Administration's (FDA) issuance of a Complete Response Letter (CRL) concerning its New Drug Application (NDA) for CUTX-101. This medication is designed to treat Menkes disease, a rare genetic disorder in pediatric patients characterized by an inability to absorb dietary copper.

FDA's Findings and Next Steps

The CRL indicates that while Sentynl's submission holds promise, certain issues must be resolved before advancing. Notably, the FDA cited concerns relating to a Current Good Manufacturing Practice (CGMP) inspection at the facility where CUTX-101 is produced. In response, Zydus has made recent submissions to address the FDA's findings from its re-inspection in September 2025, affirming the facility's compliance with CGMP standards. Now, the company is awaiting the FDA's Establishment Inspection Report (EIR).

To clarify the path forward, Sentynl intends to arrange a meeting with the FDA. This step aims to address the details specified in the CRL and discuss the potential for resubmitting the CUTX-101 NDA. Importantly, the CRL did not raise any concerns regarding the efficacy or safety data of CUTX-101, which is encouraging news for those invested in the drug's development.

The Importance of CUTX-101

Menkes disease presents a significant challenge, affecting an estimated 1 in 34,810 to 1 in 8,664 live male births. The condition, caused by mutations in the ATP7A gene responsible for copper transport, leads to severe health implications if untreated. Symptoms can include sparse or depigmented hair, seizures, developmental delays, and a high mortality rate among those who do not receive timely treatments. CUTX-101 offers hope as a subcutaneous injectable formulation that restores copper homeostasis in affected patients.

The NDA for CUTX-101 previously received FDA Priority Review status, underpinned by promising clinical efficacy results that indicated substantial improvements in the survival rates of early-treated Menkes disease patients.

Sentynl's Commitment to Patients

Matt Heck, CEO of Sentynl Therapeutics, expressed the company's unwavering dedication to the patient community. He stated, “We recognize the FDA's decision and remain dedicated to working with the Agency to clarify next steps. Our commitment to patients is unchanged. We believe in the promise of our therapy and are prepared to address the feedback and pursue resubmission promptly.” This commitment reflects Sentynl's broader mission to bring innovative solutions to those grappling with rare diseases.

About Sentynl Therapeutics and Zydus Lifesciences

Sentynl Therapeutics is known for its focus on developing therapies aimed at rare diseases. With the backing of Zydus Group, a global life sciences company, Sentynl leverages extensive resources to enhance treatment accessibility and patient outcomes. Zydus itself employs a workforce of over 29,000, including 1,500 scientists dedicated to research and development.

Through continued collaboration with regulatory agencies and dedication to compliance, Sentynl Therapeutics seeks to transform the treatment landscape for Menkes disease and similar conditions. The upcoming discussions with the FDA will be pivotal in determining the future of CUTX-101, a beacon of hope for patients facing this devastating disease.

For more detailed corporate information, visit Sentynl Therapeutics and Zydus Lifesciences.