Lundbeck has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to asedebart (Lu AG13909), Lundbeck's novel investigational anti-ACTH monoclonal antibody, for the treatment of endogenous Cushing's syndrome.

Endogenous Cushing's syndrome includes both ACTH-dependent and ACTH-independent forms. ACTH-dependent Cushing's syndrome is a rare, serious endocrine disorder caused by excess secretion of adrenocorticotropic hormone (ACTH), most commonly from a pituitary tumor (Cushing's disease) and less frequently from an ectopic ACTH-secreting tumor.

Elevated ACTH drives increased adrenal production of glucocorticoids, mineralocorticoids and androgens, disrupting normal physiological homeostasis. Of particular importance is sustained cortisol excess, which contributes to a considerable disease burden through metabolic, cardiovascular and neuropsychiatric complications and is associated with increased morbidity and mortality.

Although existing medical therapies can reduce or control elevated cortisol levels, important treatment gaps remain. Achieving and maintaining adequate disease control can be difficult, with available therapies differing in their efficacy and potentially constrained by safety and tolerability considerations.

Asedebart is a novel investigational monoclonal antibody targeting ACTH and is being developed for ACTH-dependent Cushing's syndrome. It is advancing in clinical development as a potential first-in-class treatment for rare conditions characterised by excess ACTH, with proof-of-concept trials ongoing in CD and classic Congenital Adrenal Hyperplasia (CAH) to evaluate efficacy and safety.

With this designation, asedebart continues to build momentum with regulatory authorities across rare ACTH-driven disorders. Asedebart has also received ODD in the European Union (EU) for Cushing's syndrome of endogenous origin, in addition to previous ODDs for CAH in the EU and the US, and for CAH and CD in Japan.

Tarek Samad, EVP and Head of Research and Development, Lundbeck, said, "The FDA Orphan Drug Designation is an important step for asedebart and for Lundbeck's growing commitment to rare neuroendocrine disorders. ACTH-dependent Cushing's syndrome can be a devastating condition for patients, with long-term consequences that remain difficult to control despite available treatments. This milestone reflects the strength of the science behind asedebart's development to date and supports our ambition to advance innovative medicines in areas where patients continue to face significant unmet need."

Asedebart is an investigational compound that is not approved for marketing by any regulatory authority worldwide, and its efficacy and safety have not been established.