Orphan designation in the EU brings Lundbeck protocol assistance and fee reductions during development, plus ten years of market exclusivity if the drug is ultimately approved.

“Orphan designation in the European Union is an important recognition of both the unmet need in Cushing’s and the scientific rationale behind asedebart,” says Johan Luthman, Executive Vice President of R&D at Lundbeck.

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Lundbeck receives orphan drug designation in Japan 

The orphan drug designation has been granted for asedebart for the treatment of patients with congenital adrenal hyperplasia and Cushing’s disease.

Asedebart

Asedebart works by blocking ACTH from binding to melanocortin 2 receptors in the adrenal glands, thereby reducing excess secretion of glucocorticoids, mineralocorticoids and androgens. It is being developed for ACTH-dependent forms of Cushing’s syndrome, a rare endocrine disorder most often caused by pituitary or ectopic ACTH-secreting tumors, where chronic cortisol excess drives serious metabolic, cardiovascular and neuropsychiatric complications and current treatment options offer inconsistent efficacy and tolerability. The compound is currently advancing through proof-of-concept trials in both Cushing’s disease and congenital adrenal hyperplasia.