ACTH-dependent Cushing’s syndrome is a rare, serious endocrine disorder driven by excess adrenocorticotropic hormone (ACTH), most often from a pituitary tumor, leading to chronic cortisol excess with significant metabolic, cardiovascular and neuropsychiatric complications. Current medical therapies can help manage cortisol levels, but disease control remains difficult and existing options are often constrained by safety and tolerability issues.

Asedebart is a humanized monoclonal antibody that blocks ACTH from binding to the melanocortin 2 receptor in the adrenal glands, inhibiting the hormone’s downstream signalling and reducing adrenal hormone secretion. It is in clinical development as a potential first-in-class treatment for rare ACTH-driven conditions, with proof-of-concept trials ongoing in Cushing’s disease and classic congenital adrenal hyperplasia (CAH).

The US designation follows earlier orphan drug status for asedebart in the EU for Cushing’s syndrome of endogenous origin, as well as prior orphan designations for CAH in the EU and US, and for CAH and Cushing’s disease in Japan.

Tarek Samad, Executive Vice President and Head of Research & Development at Lundbeck, commented: “The FDA Orphan Drug Designation is an important step for asedebart and for Lundbeck’s growing commitment to rare neuroendocrine disorders. ACTH-dependent Cushing’s syndrome can be a devastating condition for patients, with long-term consequences that remain difficult to control despite available treatments.”