CUTX-101 is intended to treat Menkes disease. Credit: luchschenF / Shutterstock.

The US Food and Drug Administration (FDA) has accepted for filing and priority review of the new drug application (NDA) for Sentynl Therapeutics’ CUTX-101 (copper histidinate) for the treatment of Menkes disease.

Sentynl announced the acceptance with Fortress Biotech, with the former taking over complete responsibility for the therapy’s development and commercialisation from Fortress Biotech subsidiary Cyprium Therapeutics as of December 2023.

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The therapy may be eligible for a rare pediatric disease priority review voucher (PRV) if approved. Cyprium would retain ownership of this voucher, which could be used for a subsequent marketing application or sold or transferred to a third party.

Additionally, Cyprium is positioned to obtain royalties and aggregate development and sales milestones of up to $129m from Sentynl.

Fortress Biotech chairman, president and CEO and Cyprium Therapeutics chairman Lindsay Rosenwald said: “We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a programme 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 paediatric disease.”

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The NDA submission for the therapy is backed by positive topline clinical efficacy outcomes, which have shown a statistically ‘significant improvement’ in overall survival (OS) among patients when treated early with the therapy. According to the data, a nearly 80% decrease is observed in the mortality risk when compared to an untreated historical control group.

CUTX-101 has received the FDA fast track, rare pediatric disease, orphan drug, and breakthrough designations.

The European Medicines Agency (EMA) has also granted it orphan designation. The FDA, under the Prescription Drug User Fee Act (PDUFA), has set a six-month review period with a target action date of 30 June this year.

A rare genetic disorder affecting copper levels in the body, Menkes disease is caused by mutations in the ATPase Copper Transporting Alpha (ATP7A) gene.