Summary

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• This summary is based on the review of three randomized controlled trial(s). ^[1-3]
• In adults with Congenital Adrenal Hyperplasia (CAH), crinecerfont led to a 27.3% mean reduction in daily glucocorticoid dose at week 24 versus 10.3% with placebo (Least Squares Mean Difference [LSMD], -17.0 percentage points; P < 0.001); 63% of patients achieved a physiologic dose compared to 18% with placebo (P < 0.001). In children with CAH, there was an 18.0% decrease by week 28 versus a 5.6% increase with placebo (LSMD, -23.5 percentage points; P < 0.001).
• Crinecerfont reduced androstenedione levels by 299 ng/dL at week 4 in adults with CAH versus a 45.5 ng/dL increase with placebo (LSMD, -345 ng/dL; P < 0.001), and by 197 ng/dL in children versus a 71 ng/dL increase with placebo (LSMD, -268 ng/dL; P < 0.001); mean androstenedione levels were 208 ng/dL (crinecerfont) and 545 ng/dL (placebo) in children.
• In adults with 21-hydroxylase deficiency (21OHD), 14-day crinecerfont treatment reduced Adrenocorticotropic Hormone (ACTH) by 66%, 17-hydroxyprogesterone by 64%, and androstenedione by 64% with the 100 mg twice-daily regimen. Additionally, 73% of women had a ≥50% reduction in testosterone, while men experienced median reductions of 26% to 65% in androstenedione/testosterone ratios.
• In adults with CAH, the most common adverse events (AEs) were fatigue and headache. In children with CAH, the most common AEs were headache, pyrexia, and vomiting.
• In adults with 21OHD, crinecerfont was reported to be well-tolerated over a 14-day treatment period, although no specific AEs were provided.
• Crinecerfont demonstrated clinically significant reductions in glucocorticoid dose and androstenedione levels in both adults and children with CAH, with adults showing a 27.3% reduction versus 10.3% in placebo and 63% achieving physiologic doses (versus 18%; P < 0.001), and children showing an 18.0% reduction versus a 5.6% increase with placebo (LSMD, -23.5 percentage points; P < 0.001). In adults with 21OHD, it reduced ACTH by 66%, 17-hydroxyprogesterone by 64%, androstenedione by 64%, testosterone levels by ≥50% in 73% of women, and androstenedione/testosterone ratios by 26–65% in men.