FDA Grants Orphan Drug Status To Crinetics' Atumelnant For Classic Congenital Adrenal Hyperplasia

(RTTNews) - Crinetics Pharmaceuticals, Inc. (CRNX) Thursday said that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to atumelnant, the company's drug candidate for the treatment of classic congenital adrenal hyperplasia (CAH), a group of genetic disorders affecting the adrenal glands.

In January 2025, Crinetics had reported robust positive topline results from the Phase 2 TouCAHn study of atumelnant in adults with classic CAH.

Crinetics expects to start CALM-CAH Phase 3 study in adults and BALANCE-CAH Phase 2/3 study in pediatrics in the second half of this year.

Benefits of the orphan drug designation include 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.