Crinetics' atumelnant granted Orphan Drug Designation by FDA for CAH treatment.

Crinetics Pharmaceuticals announced that the FDA granted Orphan Drug Designation for atumelnant, a novel ACTH receptor antagonist, for the treatment of classic congenital adrenal hyperplasia (CAH). Atumelnant is the first small molecule ACTH receptor antagonist in clinical development and aims to restore normal adrenal androgen levels and reduce glucocorticoid supplementation to physiologic levels. The designation underscores the significant unmet need in CAH treatment.

Crinetics Pharmaceuticals (Nasdaq: CRNX) has received a significant boost in its development efforts with the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation (ODD) for its drug candidate atumelnant. The designation is for the treatment of classic congenital adrenal hyperplasia (CAH), a rare genetic disorder affecting fewer than 200,000 people in the U.S.

Atumelnant, the first and only small molecule ACTH receptor antagonist in clinical development, aims to address the unmet needs in CAH treatment. The drug candidate demonstrated impressive results in a Phase 2 trial, showing up to 80% mean reduction in androstenedione, a key biomarker, and meaningful improvements in clinical symptoms. Crinetics plans to initiate Phase 3 studies in adults and Phase 2/3 studies in pediatrics in the second half of 2025.

The Orphan Drug Designation provides substantial benefits, including potential fee exemptions, financial incentives for clinical development, and seven years of market exclusivity upon approval. These benefits underscore the FDA's recognition of the significant unmet need in CAH treatment and the potential of atumelnant to transform the management of this genetic disorder.

Atumelnant's unique mechanism of action as a selective melanocortin type 2 receptor (MC2R) antagonist on the adrenal gland addresses the underlying pathophysiology of CAH more directly than current treatments. The Phase 2 TouCAHn trial demonstrated substantial, rapid, and sustained reductions in key biomarkers and clinical improvements, including resumption of menses and reduction of adrenal size.

Crinetics' ambitious Phase 3 program aims to demonstrate normalization of adrenal androgens and reduction of glucocorticoid supplementation to physiologic levels. This dual approach directly addresses the core treatment challenges in CAH, controlling excess androgens while avoiding the significant side effects of supraphysiologic glucocorticoid therapy.

With the FDA's Orphan Drug Designation, Crinetics is well-positioned to advance atumelnant through its clinical trials and potentially revolutionize the treatment paradigm for CAH. The company's focus on developing innovative therapies for endocrine diseases and endocrine-related tumors continues to attract investor interest.

References:
[1] https://www.stocktitan.net/news/CRNX/crinetics-receives-fda-orphan-drug-designation-for-atumelnant-in-the-spad0ssrtbs2.html