FDA Accepts and Prioritizes Crinecerfont Applications for CAH Patients

Neurocrine Biosciences, Inc. has announced that the U.S. Food and Drug Administration (FDA) has accepted their New Drug Applications (NDA) for crinecerfont, a drug aimed at treating classic congenital adrenal hyperplasia (CAH) in patients of all ages. Crinecerfont could become the first new treatment option for CAH in 70 years, offering a novel therapeutic approach for this serious endocrine disorder. The FDA has scheduled Prescription Drug User Fee Act (PDUFA) target action dates for December 29 and December 30, 2024, indicating when it plans to complete its reviews.

The FDA’s Priority Review designation for crinecerfont underscores the significant unmet medical need for new CAH treatments. This designation shortens the FDA’s review period by four months. Should crinecerfont be approved, Neurocrine Biosciences will activate its Rare Pediatric Disease Designation Priority Review Voucher, initially granted in September 2020.

Neurocrine Biosciences’ Chief Medical Officer, Eiry W. Roberts, M.D., emphasized the urgency for new treatments for CAH, noting the compelling efficacy and safety results of crinecerfont. The drug has previously received Orphan Drug and Breakthrough Therapy designations from the FDA. These designations bring benefits such as market exclusivity, tax credits, and exemption from certain fees. The Breakthrough Therapy designation, awarded based on promising Phase 3 trial data, aims to expedite the drug’s development and review process.

The CAHtalyst™ Phase 3 studies provided the clinical data supporting the crinecerfont NDAs. Results from these studies, which included both pediatric and adult patients, were published in The New England Journal of Medicine and presented at the ENDO 2024 conference. These studies focused on patients with CAH due to 21-hydroxylase deficiency, a condition that affects adrenal hormone production and can lead to severe health issues if untreated.

Current treatments for CAH involve the use of high-dose glucocorticoids to compensate for cortisol deficiency and to suppress excessive adrenal androgens. However, long-term use of these high doses can lead to significant side effects, including metabolic issues, cardiovascular disease, and psychological impacts. Crinecerfont, a selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist, offers a different mechanism of action by reducing adrenocorticotropic hormone (ACTH) and adrenal androgens without relying on glucocorticoids. This could potentially minimize the adverse effects associated with existing treatments.

Neurocrine Biosciences is a neuroscience-focused biopharmaceutical company committed to developing treatments for under-addressed neurological, neuroendocrine, and neuropsychiatric disorders. Their comprehensive portfolio includes FDA-approved treatments for various conditions and a robust pipeline of drugs in different stages of clinical development.

The potential approval of crinecerfont represents a significant advancement in the treatment of CAH, offering hope to patients who have had limited treatment options for decades. As Neurocrine Biosciences moves closer to the PDUFA dates, they continue to collaborate with the FDA to bring this innovative therapy to market.