Neurocrine Biosciences, Inc. (NASDAQ: NBIX) announced new two-year data from its Phase 3 CAHtalyst Pediatric study showing growth improvements in children and adolescents with classic congenital adrenal hyperplasia treated with CRENESSITY (crinecerfont). The data were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago.

Among pediatric patients with advanced bone age at baseline (n=24), bone age standard deviation score decreased by a mean of 1.12 after two years of treatment. A subset of nine patients demonstrated reductions greater than two standard deviations, with mean predicted adult height increasing by 4.7 cm from baseline.

A cross-sectional caregiver survey of U.S. participants from the study's open-label extension (n=29) showed universal satisfaction with CRENESSITY treatment. All respondents reported overall satisfaction with the medication and likelihood to recommend it to others with classic congenital adrenal hyperplasia. Nearly all respondents (97%) reported greater overall hope for their child's future living with the condition.

The CAHtalyst Pediatric study included 103 pediatric patients aged four to 17 years. CRENESSITY was generally well tolerated through two years of treatment, with no new safety signals observed during long-term follow-up, according to the company's statement.

CRENESSITY received approval from the U.S. Food and Drug Administration in December 2024 for use together with glucocorticoids to control androgen levels in adults and children four years and older with classic congenital adrenal hyperplasia. The medication is designed to reduce excess adrenocorticotropic hormone and adrenal androgens through a non-glucocorticoid mechanism.