Neurocrine Biosciences has received the US Food and Drug Administration (FDA) Priority Review for crinecerfont to treat patients with classic congenital adrenal hyperplasia (CAH).
CAH is a rare genetic condition that results in an enzyme deficiency, affecting the production of adrenal hormones which are essential for life.
Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed for the treatment of CAH due to 21-hydroxylase deficiency.
The US health regulator accepted the biotechnology company’s two new drug applications (NDAs) for crinecerfont as a capsule formulation and as an oral solution formulation.
If approved, crinecerfont would be the first new treatment option for CAH in 70 years, with a novel approach to the treatment of the rare endocrine disorder.
US FDA has allotted Prescription Drug User Fee (PDUFA) target action dates of 29 December and 30 December 2024 and is not planning to hold an advisory committee meeting.
Neurocrine Biosciences chief medical officer Eiry W Roberts said: “Receipt of a Priority Review reflects the FDA’s agreement that CAH is a serious condition and there is an urgent need for patients to have access to new treatments.
“Crinecerfont’s compelling efficacy results and excellent safety profile support our filing, and we look forward to working with the FDA as we head toward the PDUFA dates at the end of 2024.”
The FDA priority review for crinecerfont follows the Orphan Drug designation granted in March 2019, and the Breakthrough Therapy designation in December last year.
Through the Priority Review designation, the US FDA will advance the review timeline by four months and identify CAH as a serious condition with high unmet medical need.
The US regulatory agency will recognise crinecerfont as a new treatment that provides a significant benefit over current therapy.
Neurocrine Biosciences can activate its Rare Pediatric Disease Designation Priority Review Voucher, granted in September 2020, once crinecerfont receives FDA approval.
