adminFDA Approves New Drug ‘Crenessity’ for Treating Rare Genetic Disorder CAH
The U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences’ new drug, Crenessity, to treat classic congenital adrenal hyperplasia (CAH), a rare genetic disorder affecting hormone production in the adrenal glands. This approval offers a new option for managing the condition in adults and pediatric patients aged 4 years and older.
CAH is a condition where the adrenal glands do not produce enough cortisol, a vital hormone that helps regulate stress, while producing excess androgens, a testosterone-like hormone. To manage the condition, patients typically require higher doses of glucocorticoids, a type of steroid, to control androgen levels.
Crenessity works by reducing excessive androgen production, thereby lowering the amount of glucocorticoid treatment required. This dual-action approach helps maintain hormonal balance with less reliance on steroids.
The FDA’s approval is based on evidence from two clinical trials involving 182 adults and103 children with CAH. In the first trial, 122 adults were administered Crenessity twice daily, while 60 participants received a placebo over a 24-week period. Results showed that patients taking Crenessity reduced their daily glucocorticoid dose by 27%, while also keeping levels of androstenedione — a key androgen hormone — under control. In comparison, the placebo group only achieved a 10% reduction in glucocorticoid dosage.
The second trial evaluated 69 pediatric patients who received Crenessity twice daily and 34 who took a placebo for 28 weeks. The pediatric group taking Crenessity reduced their daily glucocorticoid dose by 18%, while the placebo group experienced a 6% increase in their glucocorticoid dosage.
This approval marks a significant development for patients living with CAH, as it offers a more effective way to manage the condition with reduced reliance on high doses of glucocorticoids. By addressing the root cause of excess androgen production, Crenessity may provide a better quality of life for those affected by this challenging disorder.
