FDA Approves Neurocrine’s Crenessity for Rare Congenital Adrenal Hyperplasia

FDA Approval:
The U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences' Crenessity (crinecerfont) for the treatment of classic congenital adrenal hyperplasia (CAH) in adults and pediatric patients aged 4 years and older35.

Breakthrough Treatment:
Crenessity is the first new treatment for CAH in 70 years, offering a paradigm-shifting approach by directly reducing excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production, allowing for glucocorticoid dose reduction25.

Clinical Trials:
The approval is based on two randomized, double-blind, placebo-controlled trials involving 182 adults and 103 children with classic CAH, demonstrating significant reductions in glucocorticoid doses and androgen levels35.

Availability:
Crenessity is expected to be commercially available in approximately one week and will be provided through PANTHERx Rare, a specialty pharmacy5.

Designations:
Crenessity received Fast Track, Breakthrough Therapy, Orphan Drug, and Priority Review designations from the FDA, highlighting its potential to address a significant unmet medical need in the rare disease community34.

Sources:

2. https://www.fiercepharma.com/pharma/neurocrine-biosciences-scores-fda-rare-genetic-adrenal-disease

3. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-congenital-adrenal-hyperplasia

4. https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-us-fda-accepts-new-drug-0

5. https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-fda-approval-crenessitytm