Crinetics Receives FDA Orphan Drug Designation for Atumelnant in Congenital Adrenal Hyperplasia
Friday, August 22, 2025
Crinetics Pharmaceuticals 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 being developed for the treatment of classic congenital adrenal hyperplasia (CAH).
Atumelnant is the first small molecule ACTH receptor antagonist to enter clinical development.
In January 2025, results from the Phase 2 TouCAHn study in adults with CAH showed strong and sustained reductions in disease biomarkers, including up to an 80% mean reduction in androstenedione. The study also indicated improvements in clinical outcomes such as menstrual function and adrenal size. The company plans to initiate the CALM-CAH Phase 3 study in adults and the BALANCE-CAH Phase 2/3 paediatric study later in 2025.
CAH is a genetic disorder that disrupts cortisol production, leading to persistently high ACTH levels. This causes excess secretion of adrenal androgens and steroid precursors, which can result in reduced fertility, acne, excessive hair growth, and testicular adrenal rest tumours. Current treatment relies on long-term glucocorticoid therapy, often at higher-than-normal doses, which may lead to complications such as weight gain, diabetes, cardiovascular disease, and osteoporosis.
The FDA grants ODD status to medicines for rare diseases affecting fewer than 200,000 people in the United States.
The designation provides incentives such as exemption from certain regulatory fees, development support, and seven years of market exclusivity following approval.
Source: crinetics.com
