Neurocrine Biosciences Bags US FDA Nod Therapy Designation For Adrenal Hyperplasia

Neurocrine Biosciences, Inc. stated that it has obtained FDA Breakthrough Therapy designation for crinecerfont in congenital adrenal hyperplasia.

"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," said Eiry W. Roberts, chief medical officer, Neurocrine Biosciences. "The outstanding safety and efficacy results from the phase 3 CAHtalyst studies in paediatric and adult patients suggest that crinecerfont has the potential to represent a substantial improvement over current standard of care in CAH by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024."

Breakthrough Therapy designation is a process developed by the FDA to expedite development and review of drugs that are intended to treat a serious condition and where clinical evidence indicates that the potential drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). To learn more about Breakthrough Therapy designation, as per pharmabiz.

Congenital adrenal hyperplasia (CAH) is a series of hereditary diseases that result in an enzyme shortage that changes the production of vital adrenal hormones. A mutation that causes a deficiency of the enzyme 21-hydroxylase (21-OHD) causes approximately 95% of CAH cases. A significant deficit of this enzyme in classic CAH causes the adrenal glands to be unable to produce cortisol and, in around 75% of cases, aldosterone. If untreated, classic CAH can cause salt loss, dehydration, and even death.