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Sentnyl’s NDA for CUTX-101 Accepted by FDA for Filing and Priority Review

Has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease.

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By: Charlie Sternberg

Associate Editor, Contract Pharma

The U.S. Food and Drug Administration (FDA) has accepted Sentynl Therapeutics Inc.’s New Drug Application (NDA) for CUTX-101 for filing and Priority review.

CUTX-101 is a product candidate for the treatment of Menkes disease, 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% 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.

“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,” said Matt Heck, President & CEO of Sentynl. “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.

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