Neurocrine presents data from CAHtalyst Pediatric Phase 3 clinical study

Neurocrine Biosciences announced that it presented the CAHtalyst Pediatric Phase 3 clinical study baseline characteristics data for children and adolescents with congenital adrenal hyperplasia, or CAH, due to 21-hydroxylase deficiency enrolled in the study, along with CAHtalog Registry data assessing glucocorticoid treatment patterns in pediatric and adult patients with CAH, at the Pediatric Endocrine Society 2024 Annual Meeting in Chicago. “In 2023, Neurocrine Biosciences announced positive top-line data from the CAHtalyst Pediatric and CAHtalyst Adult Phase 3 clinical studies evaluating the efficacy, safety, and tolerability of crinecerfont in pediatric and adult patients with CAH due to 21-hydroxylase deficiency. The data from both studies supported two New Drug Applications that were submitted to the U.S. Food and Drug Administration in April 2024,” the company stated. “At baseline, many participants in the CAHtalyst Pediatric study showed clinical evidence of elevated glucocorticoid doses and adrenal androgen excess. Many exhibited obesity, advanced bone age, and early puberty, all of which can negatively impact development in childhood and adolescence, and lead to further harm in adulthood. With adrenal androgen levels elevated despite supraphysiologic glucocorticoid doses, it’s clear that there is a significant need for a new approach to treat this condition,” added Eiry W. Roberts M.D., Chief Medical Officer at Neurocrine Biosciences.