New Treatment Approved for Congenital Adrenal Hyperplasia: Daily Dose

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On December 13, 2024, we reported on on the US FDA approval of crinecerfont (Crenessity; Neurocrine Biosciences) for treatment of congenital adrenal hyperplasia (CAH) in adults and children aged 4 years and older.

The approval

Crinecerfont, a first-in-class selective corticotropin-releasing factor type 1 (CRF-1) receptor antagonist that reduces excess adrenal androgens for people with CAH due to 21-hyroxylase deficiency, is the first new therapy for CAH in 70 years. The drug will be available as capsules and oral solution and is indicated as adjunctive treatment to glucocorticoid (GC) replacement therapy for adults and children with classic CAH.

The FDA's decision was based on data from the phase 3 CAHtalyst clinical trial program, which showed participants treated with crinecerfont had reductions in supraphysiologic GC therapy and reduced androgen levels while maintaining androstenedione control.

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