CAHtalyzing Change in Congenital Adrenal Hyperplasia

This editorial discusses advances in treating congenital adrenal hyperplasia (CAH), a disorder primarily caused by 21 hydroxylase deficiency leading to adrenal insufficiency and androgen excess. Traditional high dose glucocorticoid therapy, while controlling adrenal androgens, carries significant metabolic risks and reduces life expectancy by 18 years. The CAHtalyst phase 3 trials demonstrate promising results for crinecerfont, an oral CRF receptor 1 antagonist. In adults (N=182) and children (N=103), crinecerfont enabled glucocorticoid dose reductions (27.3% vs. 10.3% placebo in adults) while maintaining androgen control (androstenedione levels). Though short term (24–28 weeks) safety was favorable, long term outcomes require validation. The findings complement earlier research on modified release hydrocortisone, offering new strategies to mitigate glucocorticoid overexposure in CAH.

This editorial discusses advances in treating congenital adrenal hyperplasia (CAH), a disorder primarily caused by 21 hydroxylase deficiency leading to adrenal insufficiency and androgen excess. Traditional high dose glucocorticoid therapy, while controlling adrenal androgens, carries significant metabolic risks and reduces life expectancy by 18 years. The CAHtalyst phase 3 trials demonstrate promising results for crinecerfont, an oral CRF receptor 1 antagonist. In adults (N=182) and children (N=103), crinecerfont enabled glucocorticoid dose reductions (27.3% vs. 10.3% placebo in adults) while maintaining androgen control (androstenedione levels). Though short term (24–28 weeks) safety was favorable, long term outcomes require validation. The findings complement earlier research on modified release hydrocortisone, offering new strategies to mitigate glucocorticoid overexposure in CAH.