Neurocrine Biosciences, Inc. presented the CAHtalyst? Pediatric Phase 3 clinical study baseline characteristics data for children and adolescents with congenital adrenal hyperplasia (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.

These data demonstrate the limitations of current CAH treatment approaches in pediatric patients, comorbidities associated with the condition and/or current treatment, including obesity, advanced bone age, and early puberty, and the difficulty in managing the disease effectively as CAH patients age into adults. These new data were presented at the Pediatric Endocrine Society 2024 Annual Meeting in Chicago. Baseline characteristics of the subjects who enrolled in the CAHtalyst Pediatric Phase 3 study were presented (Poster# 56).

The study enrolled 103 subjects 4 to 17 years of age with a medically confirmed diagnosis of CAH due to 21-hydroxylase deficiency, with 52% male, mean age 12 years old, and majority in Tanner stages 3-5. There was evidence indicating inadequate adrenal androgen control in many of these patients despite supraphysiologic glucocorticoid dosing. At baseline, more than a third of the participants reported comorbidities of advanced bone age, early puberty, and obesity. Hirsutism (excessive hair growth, 12%) and irregular menses (12%) were reported in females, and testicular adrenal rest tumors were identified in more than a third of males.

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.