Crinetics Pharmaceuticals, Inc. announced on August 21, 2025, that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for atumelnant. Atumelnant is a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH).
Atumelnant is the first and only small molecule ACTH receptor antagonist in clinical development. The ODD status provides significant benefits, including exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. if the treatment is approved.
This designation follows robust positive topline results from the Phase 2 TouCAHn trial in January 2025, which demonstrated substantial, rapid, and sustained reductions in key biomarkers, including up to an 80% mean reduction in androstenedione. Crinetics expects to initiate the CALM-CAH Phase 3 study in adults and the BALANCE-CAH Phase 2/3 study in pediatrics in the second half of 2025.
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