Valby, Denmark – – September 16, 2026 -- The U.S. Food and Drug Administration has granted Orphan Drug Designation to asedebart (Lu AG13909), Lundbeck's investigational anti-ACTH monoclonal antibody, for the treatment of endogenous Cushing's syndrome.
FDA designation adds U.S. market exclusivity potential for Lundbeck's rare disease pipeline
The designation covers drugs and biologics intended for rare diseases and can provide tax credits for qualified clinical testing, exemption from certain FDA application fees, and up to seven years of market exclusivity if the drug is approved for the designated indication.
Asedebart targets ACTH to address treatment gaps in Cushing's disease
Endogenous Cushing's syndrome is driven in most cases by excess adrenocorticotropic hormone (ACTH), most commonly from a pituitary tumor known as Cushing's disease. Chronic cortisol excess linked to the condition causes metabolic, cardiovascular and neuropsychiatric complications and is associated with increased morbidity and mortality. Current medical therapies face limitations in efficacy, safety and tolerability, according to Lundbeck.
Asedebart is a humanized monoclonal antibody that blocks ACTH from binding to the melanocortin 2 receptor in the adrenal glands, inhibiting downstream secretion of glucocorticoids, mineralocorticoids and androgens. A proof-of-concept trial, registered as NCT06471829 under the name BalanCeD, is currently evaluating the antibody's efficacy and safety in adult patients with Cushing's disease. Separate proof-of-concept trials are also underway in classic congenital adrenal hyperplasia (CAH).
Regulators in three regions have now granted orphan status across ACTH-driven disorders
The FDA's Cushing's syndrome designation follows earlier orphan drug designations granted to asedebart for CAH in both the European Union and the United States, and for CAH and Cushing's disease in Japan. The compound also holds EU orphan designation for Cushing's syndrome of endogenous origin.
"The FDA Orphan Drug Designation is an important step for asedebart and for Lundbeck's growing commitment to rare neuroendocrine disorders," said Tarek Samad, Executive Vice President and Head of Research & Development at Lundbeck.
Asedebart remains an investigational compound not approved for marketing by any regulatory authority worldwide, and its efficacy and safety have not been established.