Danish biopharmaceutical company H. Lundbeck A/S (CPH: HLUN-B) said on Monday it has received European Commission orphan designation for asedebart (Lu AG13909), its investigational anti-ACTH monoclonal antibody for Cushing's syndrome of endogenous origin.

Cushing's syndrome of endogenous origin includes ACTH-dependent and ACTH-independent forms. ACTH-dependent disease is typically caused by excess adrenocorticotropic hormone (ATCH) from a pituitary tumour or, less commonly, an ectopic ACTH-secreting tumour.

Chronic cortisol excess drives metabolic, cardiovascular and neuropsychiatric complications and is associated with increased morbidity and mortality. Current therapies can help manage cortisol levels, but disease control is often difficult and treatment options may be constrained by variable efficacy, safety and tolerability.

Asedebart is a humanised monoclonal antibody that blocks ACTH binding to the melanocortin 2 receptor in the adrenal glands. It is in clinical development as a potential first-in-class therapy, with proof-of-concept trials under way in Cushing's disease and classic congenital adrenal hyperplasia. The compound has previously received orphan designations for congenital adrenal hyperplasia in the European Union and the United States, and for congenital adrenal hyperplasia and Cushing's disease in Japan.

In the European Union, orphan designation supports development of medicines for rare, life-threatening or chronically debilitating conditions and may provide incentives such as protocol assistance, fee reductions and, if approved, ten years of market exclusivity.

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