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).

Crinetics Pharmaceuticals 已获得美国食品药品监督管理局（FDA）授予的孤儿药资格（ODD），用于其正在开发的每日一次口服促肾上腺皮质激素（ACTH）受体拮抗剂 atumelnant，该药物用于治疗经典型先天性肾上腺增生症（CAH）。

Atumelnant is the first small molecule ACTH receptor antagonist to enter clinical development.

阿图美兰是首个进入临床开发的小分子ACTH受体拮抗剂。

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..

2025年1月，针对成年CAH患者的二期TouCAHn研究结果显示，疾病生物标志物显著且持续减少，其中雄烯二酮的平均降幅高达80%。研究还表明，月经功能和肾上腺大小等临床结果有所改善。公司计划在2025年下半年启动针对成人的三期CALM-CAH研究以及针对儿童的二期/三期BALANCE-CAH研究。

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..

先天性肾上腺皮质增生症（CAH）是一种遗传性疾病，会破坏皮质醇的生成，导致ACTH水平持续偏高。这会引起肾上腺雄激素和类固醇前体的过量分泌，可能导致生育能力下降、痤疮、多毛症以及睾丸肾上腺残留肿瘤。目前的治疗方法依赖于长期的糖皮质激素治疗，通常使用高于正常剂量的药物，但这可能会引发诸如体重增加、糖尿病、心血管疾病和骨质疏松等并发症。

The FDA grants ODD status to medicines for rare diseases affecting fewer than 200,000 people in the United States.

美国食品药品监督管理局（FDA）授予用于在美国影响不到20万人的罕见疾病的药物ODD地位。

The designation provides incentives such as exemption from certain regulatory fees, development support, and seven years of market exclusivity following approval.

该指定提供了诸如免除某些监管费用、开发支持以及在批准后七年的市场独占期等激励措施。