Neurocrine Biosciences, Inc. announced new results from subgroup analyses of the Phase 3 CAHtalyst Pediatric study, demonstrating that pediatric patients with classic congenital adrenal hyperplasia (CAH) consistently maintained or improved their androstenedione levels with CRENESSITY while reducing glucocorticoid dosing across all analyzed subgroups. These data will be presented at the 2025 Joint Congress of European Society for Paediatric Endocrinology and the European Society of Endocrinology.
Overproduction of androstenedione, a key adrenal androgen, in pediatric CAH patients can lead to abnormal growth and development, premature puberty, and various developmental challenges. Historically, high levels of androstenedione were treated solely with high-dose glucocorticoids (GCs), which are often associated with side effects and complications.
The CAHtalyst Pediatric study included 103 patients, randomized to receive either CRENESSITY or placebo for 28 weeks. The ability to reduce GC doses while simultaneously maintaining or improving androgen levels represents a significant advancement in CAH management, suggesting CRENESSITY may improve long-term outcomes across diverse patient characteristics.
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