Neurocrine receives FDA Breakthrough Therapy designation for crinecerfont

Neurocrine Biosciences announced it received Breakthrough Therapy designation from the U.S. FDA 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 Roberts, Chief Medical Officer, Neurocrine Biosciences. “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 CAH by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024.” At the company’s Analyst Day, Neurocrine Biosciences provided an update on its R&D portfolio and strategy, including the company’s focus on building breadth and depth across therapeutic areas and modalities with key focus areas in VMAT2 inhibition, CRF antagonism, muscarinic agonism to antagonism, and gene therapy. With a diversified early-stage portfolio spanning a range of modalities including small molecules and biologics, and a growing pre-clinical and development candidate portfolio, Neurocrine is uniquely positioned to advance a steady flow of innovative clinical candidates to patients across its neuroscience-focused therapeutic areas of interest. The company remains on-track to advance two gene therapies into the clinic in 2025, and anticipates at least 20 development candidates by 2027.