San Diego, USA | May 1, 2026
Neurocrine Biosciences has reported new two-year Phase 3 clinical data for CRENESSITY® (crinecerfont) demonstrating durable hormone control, reduced glucocorticoid exposure, and meaningful clinical improvements in pediatric patients with classic congenital adrenal hyperplasia (CAH). These long-term results reinforce the therapy’s potential to redefine treatment standards in rare endocrine disorders by improving disease control while minimizing steroid-related complications.
Two-Year Clinical Data Demonstrates Sustained Efficacy
The updated findings from the Phase 3 CAHtalyst® Pediatric study show that CRENESSITY delivers sustained reductions in key hormonal markers, including adrenocorticotropic hormone (ACTH) and 17-hydroxyprogesterone (17-OHP), over a 24-month period. Importantly, these improvements were achieved alongside significant reductions in glucocorticoid (GC) dosing, allowing patients to move toward more physiologic hormone replacement levels.
The study included 86 pediatric participants aged 4 to 17 years, with results indicating continued disease control even as steroid exposure was reduced. This dual benefit addresses a long-standing challenge in CAH management, where excessive steroid use often leads to serious long-term complications.
Meaningful Improvements in Metabolic and Clinical Outcomes
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Beyond hormonal control, CRENESSITY demonstrated clinically meaningful improvements in key health outcomes associated with CAH and long-term steroid use. Approximately 60% of overweight or obese patients experienced improvements in body mass index (BMI), while 61% of patients with baseline insulin resistance were no longer insulin resistant after two years of treatment.
Additional benefits included improvements in acne severity, androgen levels, and metabolic markers, highlighting the therapy’s broader impact on patient quality of life and long-term health outcomes. These findings underscore the importance of reducing androgen excess and minimizing glucocorticoid exposure in pediatric populations.
Safety Profile and Long-Term Treatment Potential
CRENESSITY was generally well tolerated, with no new safety signals identified over the two-year study period, and a high patient retention rate exceeding 80%, supporting its long-term usability. The therapy works as a corticotropin-releasing factor type 1 (CRF1) receptor antagonist, reducing ACTH levels and subsequently lowering androgen production through a non-glucocorticoid mechanism.
This innovative approach enables effective disease control without the risks associated with high-dose steroid therapy. The data build upon previous one-year results and support CRENESSITY’s role as a transformative treatment option in pediatric endocrinology.
Advancing Rare Disease Treatment and Clinical Practice
Congenital adrenal hyperplasia is a rare genetic disorder characterized by impaired cortisol production and excess androgen levels, often requiring lifelong management. Traditional treatment strategies rely heavily on supraphysiologic glucocorticoid doses, which can lead to metabolic, cardiovascular, and developmental complications. CRENESSITY offers a targeted therapeutic alternative, enabling clinicians to better balance hormone control and safety. These results are expected to inform clinical practice, support regulatory discussions, and further strengthen the role of innovative therapies in rare disease management.
Source: Neurocrine Biosciences press release
