Zydus's Sentynl Therapeutics pediatric Menkes disease drug flagged by USFDA
Solana Beach: Sentynl Therapeutics, Inc., a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd., has announced that the U.S. Food and Drug Administration (USFDA) has issued a Complete Response Letter (CRL) relating to its New Drug Application (NDA) for copper histidinate (CUTX-101), intended to treat Menkes disease in pediatric patients.
The USFDA provided findings within the CRL that Sentynl and Zydus Lifesciences will need to address to clarify the path forward. Specifically, the USFDA mentioned a CGMP inspection of the facility where CUTX101 is manufactured. Zydus recently provided responses to USFDA’s September 2025 re-inspection demonstrating the facility’s CGMP compliance and is awaiting USFDA’s Establishment Inspection Report (EIR).
Sentynl will request a meeting with USFDA to discuss the CRL and resubmission of the CUTX-101 NDA. The CRL did not cite any other approvability concerns, nor did it identify any deficiencies in CUTX101’s efficacy and safety data.
“We recognize the USFDA’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,” said Matt Heck, CEO, Sentynl.
Menkes disease is a rare X-linked recessive pediatric genetic disease that impacts an estimated 1 in 34,810 to as high as 1 in 8,664 live male births. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier.
CUTX101 is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease. The CUTX-101 NDA was initially granted Priority Review by the FDA and is supported by positive topline clinical efficacy results for CUTX-101, demonstrating significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101.
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