Sentynl Therapeutics, Inc., a US-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd., and Fortress Biotech, Inc. has announced that the US Food and Drug Administration (FDA) has accepted for filing and priority review of Sentynl’s New Drug Application (NDA) for CUTX-101, the product candidate for the treatment of Menkes disease.

Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. Recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births.

Sentynl’s NDA is supported by positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, with a nearly 80 percent reduction in the risk of death compared to an untreated historical control cohort. Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort.

Commenting on the development, Punit Patel, President and CEO, Zydus Americas said, “The NDA acceptance of CUTX-101 marks an important step towards our vision to transform lives and meaningfully impact patients, caregivers, and the rare disease community at large. We remain committed to providing access to path-breaking discoveries that can address unmet needs globally, as evident in the potential impact of CUTX-101 on Menkes disease patients and their families.”

Matt Heck, President & Chief Executive Officer of Sentynl remarked, “Menkes disease presents a difficult journey for patients and their caregivers, as ATP7A mutations impact the transport of copper to a range of organs and systems, such as the lungs, brain and heart. With no known cure or current FDA-approved treatments, death typically occurs between 2 to 3 years of age. We are eager for the FDA to review our application for CUTX-101, which has the potential to be the first FDA-approved therapy for this devastating condition.”

CUTX-101 was granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations by the FDA and Orphan Designation by the European Medicines Agency (EMA). Under the Prescription Drug User Fee Act (PDUFA), the FDA set a six-month period with a target action date of June 30, 2025.

In December 2023, Sentynl assumed full responsibility for the development and commercialisation of CUTX-101 from Cyprium Therapeutics, a Fortress subsidiary company focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders.

Lindsay A. Rosenwald, MD, Chairman, President and Chief Executive Officer of Fortress and Chairman of Cyprium, “We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a program that Cyprium advanced and proudly supports. The drug has a demonstrated safety and efficacy profile for the treatment of Menkes disease and, if approved, CUTX-101 will fill a significant unmet need for children suffering from this rare, fatal pediatric disease.”