San Diego, USA – April 22, 2026

In a major advancement for endocrine disorder management, Neurocrine Biosciences has unveiled new two-year clinical data demonstrating that CRENESSITY® (crinecerfont) delivers sustained glucocorticoid dose reductions while maintaining androgen control in adults with classic congenital adrenal hyperplasia (CAH). The findings, presented at a leading endocrinology conference, highlight a potential shift in long-term disease management, addressing the critical burden associated with chronic high-dose steroid therapy.

Durable Efficacy with Reduced Steroid Burden

The Phase 3 CAHtalyst Adult study revealed that approximately 70% of patients achieved physiologic-range glucocorticoid dosing after two years of treatment with crinecerfont, marking a significant improvement over traditional approaches. Notably, patients experienced an average 38% reduction in glucocorticoid exposure, a key milestone given the long-standing reliance on supraphysiologic steroid doses to control disease symptoms.

Equally important, these reductions were achieved without compromising androgen control, a critical therapeutic objective in CAH management. Around 75% of patients previously treated with dexamethasone transitioned to steroid regimens closer to physiological norms, highlighting CRENESSITY’s ability to enable safer, more sustainable treatment strategies.

The study further demonstrated that long-term treatment was well tolerated, with over 80% patient retention at two years and no new safety signals observed, reinforcing confidence in its chronic use potential.

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Addressing Risks of Traditional CAH Treatments

Congenital Adrenal Hyperplasia is a rare genetic disorder characterized by impaired cortisol production and excess androgen levels, often requiring lifelong treatment. Historically, patients have depended on high-dose glucocorticoids, which are associated with serious long-term complications, including cardiometabolic disease, osteoporosis, mental health disorders, and reduced quality of life.

CRENESSITY introduces a novel non-glucocorticoid mechanism, acting as a corticotropin-releasing factor type 1 (CRF1) receptor antagonist, which directly reduces adrenocorticotropic hormone (ACTH) levels and downstream androgen production. This approach allows clinicians to lower steroid doses while maintaining hormonal balance, addressing one of the most pressing unmet needs in CAH care.

Importantly, clinical data confirmed that reductions in glucocorticoid dosing did not lead to worsening of androstenedione levels, demonstrating that efficacy is preserved even as treatment toxicity is minimized.

Clinical Impact and Future Treatment Landscape

The newly presented data reinforce CRENESSITY’s position as a potential new standard of care for CAH, particularly in adult patients requiring long-term disease management. The therapy, already approved by the U.S. FDA in December 2024, continues to demonstrate durable clinical benefit and safety in extended use, strengthening its commercial and therapeutic outlook.

Beyond efficacy, the study underscores a broader clinical shift toward precision endocrine therapies that target disease mechanisms directly rather than relying solely on hormone replacement strategies. By enabling physiologic glucocorticoid dosing, CRENESSITY may significantly reduce lifetime treatment burden, improve adherence, and enhance patient outcomes.

Ongoing and future analyses from the CAHtalyst program are expected to further validate these findings across additional endpoints, including cardiometabolic outcomes and long-term safety, offering deeper insights into the therapy’s real-world impact.

As healthcare providers increasingly prioritize risk reduction and quality-of-life improvements, CRENESSITY stands out as a transformative innovation in the treatment of rare endocrine disorders, signaling a new era in CAH therapeutic development.

Source: Neurocrine Biosciences press release