FDA approves CRENESSITY: A breakthrough treatment from Neurocrine Biosciences for classic congenital adrenal hyperplasia

Neurocrine Biosciences has announced US Food and Drug Administration (FDA) approval of CRENESSITY, marking a significant milestone in the treatment of classic congenital adrenal hyperplasia (CAH). As the first novel therapy in over seven decades, CRENESSITY represents a paradigm shift, offering patients a potent, targeted treatment to manage adrenal hormone imbalance with fewer glucocorticoid-related side effects.

Why CRENESSITY Is a Game-Changer

CRENESSITY is the first treatment to address classic congenital adrenal hyperplasia through a non-glucocorticoid mechanism, directly targeting corticotropin-releasing factor type 1 receptors (CRF1). This approach reduces excess adrenal androgen production and adrenocorticotropic hormone (ACTH), allowing patients to decrease their reliance on high-dose glucocorticoids.

Approved for patients aged four and older, CRENESSITY comes in capsule and oral solution forms, with flexible dosing tailored to individual needs. Importantly, CRENESSITY enables more physiologic glucocorticoid dose reduction, improving long-term health outcomes for CAH patients.

Clinical Backing: Largest CAH Trials to Date

The FDA approval of CRENESSITY is based on robust clinical trial data from Neurocrine’s Pediatric Phase 3 and Adult Phase 3 CAHtalyst studies. These represent the largest-ever trials conducted for classic congenital adrenal hyperplasia, demonstrating significant reductions in adrenal androgen production and enabling lower glucocorticoid doses.

In children: CRENESSITY decreased androstenedione and 17-hydroxyprogesterone (17-OHP) levels by up to 12 and 4 times, respectively, compared to placebo, while maintaining effective androgen control.

In adults: Patients achieved glucocorticoid dose reduction in the physiologic range, with significant improvements in androstenedione and 17-OHP levels.

Safety Profile and Side Effects

Both Pediatric Phase 3 and Adult Phase 3 trials highlighted CRENESSITY’s tolerability, with no treatment-related serious adverse events. Temporary and mild-to-moderate side effects such as headache, fatigue, and abdominal discomfort were observed.

However, rare occurrences of adrenal crises were noted in adult patients, underscoring the importance of monitoring glucocorticoid replacement therapy during treatment.

Commercial Availability and Support Programs

CRENESSITY will be commercially available within a week, distributed via PANTHERx Rare, a specialty pharmacy. Neurocrine Biosciences has also launched Neurocrine Biosciences CRENESSITY Access Support, offering comprehensive assistance to help patients navigate insurance and secure financial aid, ensuring affordability—most patients will pay $10 or less monthly.

Expert Insights and Community Impact

Kyle W. Gano, CEO of Neurocrine Biosciences, emphasized that CRENESSITY’s approval is a testament to decades of research and collaboration with the CAH community. Dina Matos, Executive Director of the CARES Foundation, highlighted the therapy’s potential to improve quality of life by reducing the burden of high-dose steroid regimens, a challenge long faced by individuals managing classic congenital adrenal hyperplasia.

With its approval, CRENESSITY ushers in a new era for classic congenital adrenal hyperplasia management, offering a safer and more effective alternative to conventional treatments. Its novel mechanism, backed by extensive clinical trial data, promises to transform the lives of CAH patients worldwide.