UK—The European Commission (EC) has granted orphan designation to Lundbeck’s investigational monoclonal antibody asedebart (Lu AG13909) for the treatment of endogenous Cushing’s syndrome.
The designation recognizes Asedebart as a potential treatment for a rare and debilitating endocrine disorder for which patients continue to face significant unmet medical needs.
Rare endocrine disorder
Cushing’s syndrome of endogenous origin occurs when the body produces excessive amounts of cortisol and other steroid hormones.
In most cases, the condition results from excessive production of adrenocorticotropic hormone (ACTH), usually caused by a pituitary tumour known as Cushing’s disease.
Less commonly, ectopic tumours can produce ACTH and trigger the condition.
Excessive ACTH stimulates the adrenal glands to produce increased levels of cortisol.
This hormonal imbalance can cause substantial health problems, including metabolic and cardiovascular complications as well as neuropsychiatric effects.
Although existing medicines can help control cortisol production, achieving sustained disease control can be difficult.
In addition, some available treatments have limitations related to efficacy, safety and tolerability, highlighting the need for new therapeutic approaches.
Targeted approach to hormone signalling
Lundbeck developed asedebart as a humanised monoclonal antibody that targets ACTH.
The antibody is designed to prevent ACTH from binding to the melanocortin 2 receptor in the adrenal glands.
By blocking this interaction, asedebart aims to disrupt the hormonal signalling pathway that drives steroid hormone production.
The approach is intended to reduce the secretion of glucocorticoids, mineralocorticoids and androgens.
Lundbeck is currently evaluating the treatment in proof of concept clinical trials involving patients with Cushing’s disease and classic congenital adrenal hyperplasia (CAH).
The studies are assessing the safety and efficacy of asedebart in these conditions.
Regulatory recognition
Johan Luthman, executive vice president of research and development at Lundbeck, said the EU orphan designation recognises the significant unmet need among people living with Cushing’s syndrome and the scientific rationale supporting asedebart.
He said the programme reflects Lundbeck’s focus on developing targeted therapies for neuroendocrine and rare disorders.
The latest designation adds to a series of regulatory recognitions for asedebart.
The medicine has previously received orphan designation for classic congenital adrenal hyperplasia in the European Union, the United States and Japan.
Japan has also granted orphan designation for its development in Cushing’s disease.
Asedebart remains an investigational medicine and has not been approved for marketing in any country.
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