The FDA approved crinecerfont (Crenessity), for use with glucocorticoids to control androgen levels in patients aged 4 or older with classic congenital adrenal hyperplasia (CAH)—the drug works by reducing excessive adrenal androgen production.

Two randomized, double-blind, placebo-controlled trials supported the approval. In the adult trial, following an initial 4-week period, patients receiving twice-daily crinecerfont demonstrated a 27% reduction in daily glucocorticoid dosing while maintaining androstenedione control over 24 weeks, compared with a 10% reduction in the placebo group. The pediatric trial showed an 18% reduction in glucocorticoid requirements over 28 weeks in the treatment group, while the placebo group showed a 6% dose increase.

The adult study measured change from baseline in total glucocorticoid daily dose while maintaining androstenedione control at 24 weeks. The pediatric trial measured change from baseline in serum androstenedione at week 4.

The FDA included a boxed warning for acute adrenal insufficiency risk in patients with underlying adrenal insufficiency who do not receive adequate stress-dose glucocorticoid coverage. Reported adverse effects in adults included fatigue, dizziness, and arthralgia. Pediatric patients experienced headache, abdominal pain, and fatigue.

Drug interactions were reported with enzymes involved in crinecerfont metabolism, with dose adjustment guidance provided in the prescribing information.

The drug received Fast Track, Breakthrough Therapy, Orphan Drug, and Priority Review designations from the FDA. Neurocrine Biosciences, Inc. received the approval.

Reference: Food and Drug Administration. FDA Approves New Treatment for Congenital Adrenal Hyperplasia. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-congenital-adrenal-hyperplasia