Neurocrine Biosciences, Inc. announced it will present new data from the Phase 3 CAHtalyst Pediatric study, showing lasting reductions in glucocorticoid (GC) doses in pediatric patients with classic congenital adrenal hyperplasia (CAH) who received CRENESSITY for up to one year. These data will be presented at the Pediatric Endocrine Society (PES) 2025 Annual Meeting.
The study demonstrated that for patients on CRENESSITY, adrenocorticotropic hormone, 17-hydroxyprogesterone, and androstenedione remained below baseline levels, despite substantially decreased GC doses. Results also showed improvements in clinical outcomes, including body mass index (BMI) and insulin resistance, with a mean GC dose reduction of 2.9 mg/m2/d at Week 52 for the continuous CRENESSITY group.
CRENESITY was generally well tolerated, with no adrenal crises reported in the double-blind, placebo-controlled period. These results indicate that CRENESSITY has the potential to transform the treatment landscape for children and adolescents living with classic CAH by simultaneously addressing underlying hormonal imbalances and allowing for GC dose reduction.
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