WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has approved Crenessity (crinecerfont) for use alongside glucocorticoids in managing classic congenital adrenal hyperplasia (CAH) in patients aged four and older. This marks a significant advancement for a rare genetic condition that affects hormone production and poses long-term health challenges.
Classic CAH impacts the adrenal glands, leading to insufficient cortisol production and excessive androgen levels. Patients often require high doses of glucocorticoids not only to replace cortisol but also to suppress androgens. Crenessity’s mechanism reduces androgen production, thereby lowering the required glucocorticoid dosage and enhancing treatment outcomes.
The approval is based on two rigorous clinical trials. The first involved 182 adults, with those taking Crenessity demonstrating a 27% reduction in daily glucocorticoid doses while maintaining androgen control, compared to a 10% reduction in the placebo group. A complementary study in 103 pediatric patients showed significant reductions in androgen levels after four weeks of treatment and allowed an 18% glucocorticoid dose decrease by the trial’s end, compared to an increase in the placebo group.
The treatment, however, carries warnings for potential adrenal insufficiency if stress-related glucocorticoid needs are not met. Additional considerations, such as potential side effects including fatigue and joint pain in adults, as well as headache and abdominal pain in children, are outlined in the prescribing information.
Crenessity received multiple FDA designations, including Fast Track, Breakthrough Therapy, Orphan Drug, and Priority Review, underscoring its importance in addressing an unmet medical need in rare diseases. Developed by Neurocrine Biosciences, Inc., this approval offers a new option for CAH patients, improving disease management and quality of life.
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