SAN DIEGO, June 15, 2026
Neurocrine Biosciences has presented new two-year data from its Phase 3 CAHtalyst® Pediatric study, demonstrating that CRENESSITY® (crinecerfont) may significantly improve growth outcomes in children and adolescents living with classic congenital adrenal hyperplasia (CAH). Presented at ENDO 2026 in Chicago, the findings showed that pediatric patients with advanced bone age at baseline experienced slowed bone age progression and improved predicted adult height, highlighting the potential of the therapy to address one of the most challenging long-term complications associated with the rare endocrine disorder.
Two-Year Data Highlights Improved Bone Age and Height Outcomes
The latest analysis evaluated a subset of growing pediatric patients enrolled in the ongoing open-label extension of the Phase 3 study. Among patients with advanced bone age at baseline, bone age standard deviation scores remained stable or improved in most participants after up to two years of CRENESSITY treatment. Researchers reported a mean reduction of 1.12 in bone age SDS, while a subset of patients demonstrated reductions exceeding two standard deviations.
Importantly, these patients experienced a mean increase of 4.7 centimeters in predicted adult height, suggesting that sustained hormonal control combined with significant reductions in glucocorticoid exposure may positively influence growth and development during childhood and adolescence. Experts noted that excess adrenal androgens in CAH can accelerate skeletal maturation, often leading to compromised adult height, making these findings particularly meaningful for pediatric disease management.
Caregiver Survey Reveals Strong Satisfaction and Future Optimism
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In addition to the clinical outcomes, Neurocrine Biosciences presented results from a caregiver-reported survey assessing long-term treatment experiences with CRENESSITY. The survey revealed 100% caregiver satisfaction, with all respondents indicating they would recommend the therapy to others managing classic CAH. Caregivers also reported increased optimism regarding their child’s future health and quality of life, with nearly all respondents expressing greater hope following treatment initiation. More than three-quarters reported reduced concern about complications associated with long-term high-dose steroid exposure, including weight gain and metabolic effects. The findings underscore the broader impact of CRENESSITY beyond biochemical control, highlighting potential improvements in patient and family confidence regarding disease management and long-term outcomes.
Potential to Reduce Steroid Burden While Maintaining Disease Control
Classic congenital adrenal hyperplasia is a rare genetic endocrine disorder characterized by impaired cortisol production and excessive androgen levels, often requiring lifelong glucocorticoid therapy. While steroids remain essential, prolonged use at supraphysiologic doses can contribute to significant complications including obesity, insulin resistance, cardiovascular disease and osteoporosis. According to Neurocrine Biosciences, previous analyses from the CAHtalyst program demonstrated that CRENESSITY enabled substantial and sustained reductions in glucocorticoid doses while maintaining androgen control over two years.
These reductions were associated with improvements in body weight, insulin resistance and body mass index, potentially lowering long-term cardiometabolic risks. Researchers also reported that CRENESSITY was generally well tolerated throughout the two-year observation period, with no new safety signals identified. As the first approved non-glucocorticoid therapy designed to control excess ACTH and adrenal androgen production in classic CAH, CRENESSITY continues to strengthen its clinical profile as a transformative treatment option for pediatric and adult patients living with this lifelong endocrine disorder.
Source: Neurocrine Biosciences press release
