Crinetics Gains FDA Orphan Drug Designation for Atumelnant in Congenital Adrenal Hyperplasia (CAH)
Friday, August 22, 2025
Crinetics Pharmaceuticals, Inc. has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for atumelnant, a once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist under development for the treatment of classic congenital adrenal hyperplasia (CAH). Atumelnant is the first small molecule ACTH receptor antagonist to progress into clinical development.
The designation follows encouraging results from the Phase 2 TouCAHn study, reported in January 2025. The trial in adults with classic CAH showed rapid and sustained reductions in key biomarkers across different doses, including up to an 80% mean decrease in androstenedione. Clinical improvements were also observed, such as resumption of menstrual cycles and reduction in adrenal size.
Based on these outcomes, Crinetics plans to advance atumelnant into further studies. The CALM-CAH Phase 3 trial in adults and the BALANCE-CAH Phase 2/3 trial in children are expected to begin enrolling participants in the second half of 2025.
CAH is a genetic condition caused by mutations that impair cortisol production. The deficiency disrupts hormonal feedback mechanisms, leading to persistently high levels of ACTH. This drives excess production of androgens such as androstenedione, which can cause reduced fertility, excessive hair growth, acne, and adrenal tumours in men. Patients require cortisol replacement, but current treatment relies on long-term glucocorticoid use at higher-than-physiological doses, often resulting in complications including obesity, diabetes, cardiovascular disease and osteoporosis.
The FDA grants ODD status to therapies intended for rare conditions affecting fewer than 200,000 people in the United States. The designation provides potential benefits including reduced regulatory fees, financial support for clinical development, and seven years of market exclusivity upon approval.
Source: globenewswire.com
