Zydus-owned Sentynl's Menkes disease drug NDA resubmission accepted by USFDA

Written By : Ruchika Sharma |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2025-12-16T13:53:03+05:30 | Updated On 16 Dec 2025 1:53 PM IST

Solana beach: Sentynl Therapeutics, a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, have announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of its New Drug Application ("NDA") for copper histidinate (CUTX-101), intended to treat Menkes disease in pediatric patients.

The resubmission has been accepted as a Class I response and as a result, the Company has received January 14, 2026 as the new PDUFA date.

"We appreciate the Agency's partnership and commitment to expeditiously reviewing our NDA resubmission," said Matt Heck, CEO, Sentynl. "The acceptance of the application brings us one step closer to a milestone for patients and families who are living with Menkes disease."

Sentynl resubmitted its revised NDA on November 14, 2025 after receiving a complete response letter (CRL) from the FDA on September 30, 2025, which cited observations regarding the manufacturing site's cGMP compliance. The CRL did not cite any other approvability concerns, nor did it identify any deficiencies in CUTX-101's efficacy and safety data, which demonstrate improvement in overall survival for Menkes disease subjects who received early treatment with the therapy.

Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment. (Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD)). The condition is characterized by distinctive clinical features, including sparse and depigmented hair ("kinky hair"), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying between 2-3 years of age. (Early clinical signs and treatment of Menkes disease - ScienceDirect). Milder versions of ATP7A mutations are associated with conditions other than Menkes Disease, such as Occipital Horn Syndrome and
ATP7A-related Distal Motor Neuropathy.

Read also: CDSCO Panel Seeks Revised Phase IV Study for Zydus' Empagliflozin, Metformin FDC

sentynl therapeuticszydus lifesciencesUSFDAcopper histidinateCUTX 101menkes disease

Ruchika Sharma

Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751

Dr. Kamal Kant Kohli

Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751