Danish biopharmaceutical company H. Lundbeck A/S (CPH: HLUN-B), which focuses on brain health, said on Friday it has received US Food and Drug Administration orphan drug designation for asedebart (Lu AG13909), its investigational anti-adrenocorticotropic hormone (ACTH) monoclonal antibody for endogenous Cushing's syndrome.

Endogenous Cushing's syndrome includes ACTH-dependent and ACTH-independent forms. ACTH-dependent disease is caused by excess adrenocorticotropic hormone, typically from a pituitary tumour. Elevated ACTH increases adrenal production of glucocorticoids, mineralocorticoids and androgens, with sustained cortisol excess driving metabolic, cardiovascular and neuropsychiatric complications.

Existing therapies can reduce cortisol levels but achieving durable disease control remains difficult, with efficacy, safety and tolerability varying across treatments. Surgery is preferred when feasible, though not all patients are eligible or achieve remission.

Asedebart targets ACTH and is in development for ACTH-dependent Cushing's syndrome, with proof-of-concept trials under way in Cushing's disease and classic congenital adrenal hyperplasia. The antibody has also received orphan designations in the European Union, the United States and Japan across ACTH-driven disorders.

US orphan drug designation may provide incentives including tax credits, fee exemptions and, if approved, seven years of market exclusivity.

Asedebart remains an investigational compound with efficacy and safety yet to be established.