FDA Grants Breakthrough Therapy Designation for Oral Congenital Adrenal Hyperplasia Medication

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The US Food and Drug Administration (FDA) granted breakthrough therapy designation for an oral nonglucorticoid medication for the treatment of congenital adrenal hyperplasia, according to a press release from the manufacturer.

Crinecerfont (Neurocrine Biosciences) is a selective corticotropin-releasing factor type 1 receptor antagonist under development to lower excess adrenal androgens for people with congenital adrenal hyperplasia due to 21-hyroxylase deficiency.

The medication met its primary and secondary endpoints in 2 phase 3 CAHtalyst trials, one assessing use of crinecerfont by children and the other by adults. In the pediatric trial, children and adolescents receiving crinecerfont had a decrease in serum androstenedione from baseline to 4 weeks. Participants receiving the medication also had a greater reduction in daily glucocorticoid at 28 weeks than placebo. In the adult trial, crinecerfont was associated with a greater reduction in daily glucocorticoid while maintaining androgen control compared with placebo. The most common adverse events in the pediatric study were headache, fever, vomiting, upper respiratory tract infection, and nasopharyngitis. Among adults, the most common adverse events were fatigue, headache, and COVID-19 infection. No serious adverse events related to crinecerfont were reported, according to the press release.

Breakthrough therapy is the latest designation granted to crinecerfont by the FDA. The medication was previously granted fast track and rare pediatric disease designations.

"We are very pleased that the FDA granted breakthrough therapy designation for crinecerfont, thus recognizing both the seriousness of congenital adrenal hyperplasia and the significant unmet need currently faced by patients and families living with this condition,” Eiry W. Roberts, MD, chief medical officer for Neurocrine Biosciences, said in a press release. "The outstanding safety and efficacy results from the phase 3 CAHtalyst studies in pediatric and adult patients suggest that crinecerfont has the potential to represent a substantial improvement over current standard of care in congenital adrenal hyperplasia by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024."

Reference: Neurocrine Biosciences Receives Breakthrough Therapy Designation from U.S. Food and Drug Administration for Crinecerfont in Congenital Adrenal Hyperplasia. News item. Neurocrine Biosciences. December 5, 2023. Accessed December 5, 2023. https://www.neurocrine.com/our-company/news-and-media/news/neurocrine-biosciences-receives-breakthrough-therapy-designation-from-u-s-food-and-drug-administration-for-crinecerfont-in-congenital-adrenal-hyperplasia/