Lundbeck has secured orphan designation from the European Commission for asedebart, its investigational anti‑ACTH monoclonal antibody being developed 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 secretion of adrenocorticotropic hormone from a pituitary tumour and less frequently from an ectopic ACTH‑secreting tumour. This hormonal excess drives overproduction of steroid hormones, resulting in homeostatic disruption and chronic cortisol elevation linked to metabolic, cardiovascular and neuropsychiatric complications.
Current medical therapies can help manage cortisol excess. However unmet need remains, with disease control often limited by variable efficacy, safety and tolerability.
Asedebart is designed to target ACTH and is being developed for ACTH‑dependent forms of Cushing’s syndrome. It is progressing in clinical development as a potential first‑in‑class therapy for rare ACTH‑driven conditions, with proof‑of‑concept trials under way in Cushing’s disease and classic congenital adrenal hyperplasia.
The new designation adds to existing orphan recognitions for congenital adrenal hyperplasia in the European Union and United States, and for congenital adrenal hyperplasia and Cushing’s disease in Japan.
Johan Luthman, Executive Vice President, R&D, Lundbeck, said: “Orphan designation in the European Union is an important recognition of both the unmet need in Cushing’s and the scientific rationale behind asedebart.”
He added: “The asedebart program is a good representation of the type of strong targeted mechanism programs we like to advance in Lundbeck. The orphan designation in Cushing’s syndrome for asedebart is also another example of a breakthrough innovation pipeline in neuroendocrine and rare disorders, where several programs have been granted a number of special regulatory designations in recent years.”
In the European Union, orphan designation supports development of medicines for rare, life‑threatening or chronically debilitating diseases. Designated therapies may receive incentives including protocol assistance, fee reductions and, if approved, ten years of market exclusivity.
