Neurocrine (NBIX) announced new results from subgroup analyses of the Phase 3 CAHtalyst Pediatric study. The analyses showed that, consistently across all of the different subgroups analyzed, pediatric patients with classic congenital adrenal hyperplasia, or CAH, maintained or improved their androstenedione levels with Crenessity – crinecerfont – while reducing glucocorticoid dosing. Overproduction of androstenedione, a key adrenal androgen, in pediatric patients with congenital adrenal hyperplasia can lead to abnormal growth and development, premature puberty and various developmental challenges. For decades, high levels of androstenedione were treated with glucocorticoids only. Across all subgroups, Crenessity enabled reduction of GC doses while maintaining or improving A4 levels. Crenessity was generally well tolerated in the CAHtalyst Pediatric study. The most common adverse reactions were headache, abdominal pain, fatigue, nasal congestion and nosebleed. These results suggest that Crenessity may improve long-term outcomes in patients across various subgroups.
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