- In January 2025, Crinetics Pharmaceuticals, Inc. announced positive topline results from an open-label, Phase 2 study of atumelnant, an investigational, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist. The study focused on evaluating atumelnant for the treatment of classic congenital adrenal hyperplasia (CAH) and ACTH-dependent Cushing’s syndrome
- In January 2025, Neurocrine Biosciences, Inc. announced the publication of a supplement in The Journal of Clinical Endocrinology & Metabolism (JCEM), sponsored by the company, focused on Classic Congenital Adrenal Hyperplasia (CAH). Titled "Challenges and Opportunities in the Management of Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Throughout the Lifetime," the supplement features eight review articles that offer a thorough examination of the clinical, psychosocial, treatment-related, and daily challenges faced by individuals living with classic CAH
- In December 2024, the U.S. Food and Drug Administration approved Crenessity (crinecerfont) for use in combination with glucocorticoids (steroids) to manage androgen (testosterone-like hormone) levels in adults and pediatric patients aged 4 years and older with classic congenital adrenal hyperplasia (CAH)
- In December, 2024, Spruce Biosciences, Inc. announced the topline results from its CAHmelia-204 study of tildacerfont in adult CAH and the CAHptain-205 study of tildacerfont in both adult and pediatric patients with Congenital Adrenal Hyperplasia. The CAHmelia-204 study was a Phase 2b, randomized, double-blind, placebo-controlled trial that assessed the safety and efficacy of tildacerfont in reducing supraphysiologic glucocorticoid usage in 100 adults with classic CAH
- In September 2024, BridgeBio Pharma, Inc. announced the topline results from the Phase 1/2 open-label ADventure study, which is evaluating BBP-631, the company's experimental adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia (CAH)
