Neurocrine Biosciences Unveils Significant Findings on CRENESSITY for Congenital Adrenal Hyperplasia Treatment

Neurocrine Biosciences Unveils Groundbreaking Data on CRENESSITY

Neurocrine Biosciences, Inc. has recently announced pivotal findings from its Phase 3 studies related to CRENESSITY™, a drug formulated to treat classic congenital adrenal hyperplasia (CAH). Presented at the 2025 American Association of Clinical Endocrinology Annual Meeting in Orlando, these findings provide critical insights into the effectiveness of CRENESSITY in managing hormone levels for both pediatric and adult patients suffering from this condition.

Understanding Congenital Adrenal Hyperplasia (CAH)

Congenital adrenal hyperplasia is a genetic disorder that leads to deficiencies in specific enzymes responsible for producing vital adrenal steroids, particularly cortisol. The resultant hormonal imbalance can pose severe health risks, including salt-wasting crises and virilization. Traditionally, treatment has relied on high-dose glucocorticoids. However, excessive use of glucocorticoids can lead to numerous complications, including metabolic disorders and psychological effects, making the need for alternative therapies evident.

Key Findings of the CAHtalyst Studies

The CAHtalyst studies aimed to assess the safety and efficacy of CRENESSITY in both adult and pediatric populations. The results indicated that a significant majority of pediatric participants taking CRENESSITY achieved either a reduction in glucocorticoid doses or normalization of androstenedione levels—key indicators of adrenal function.

For instance, 90% of the pediatric participants on CRENESSITY were able to achieve this threshold compared to only 21% on placebo. Notably, none of the placebo group achieved physiological glucocorticoid dosing, a stark contrast to 30% of the CRENESSITY group.

The adult study also yielded compelling results. Male patients receiving CRENESSITY displayed significant improvements in their reproductive hormone levels. By the end of the study, approximately 65% had normalized levels of luteinizing hormone, compared to just 22% in the placebo group at Week 24.