Lundbeck has received orphan designation from the European Commission for asedebart (Lu AG13909), an experimental monoclonal antibody being developed to treat Cushing’s syndrome of endogenous origin.
Asedebart is designed to target adrenocorticotropic hormone (ACTH), which plays an important role in regulating hormone production in the adrenal glands. Lundbeck is developing the drug mainly for ACTH-dependent forms of Cushing’s syndrome.
Cushing’s syndrome is a rare and serious endocrine disorder caused by prolonged exposure to high levels of cortisol and other steroid hormones. In ACTH-dependent cases, the condition is usually caused by a pituitary tumor, known as Cushing’s disease, or less commonly by a tumor elsewhere in the body that produces ACTH.
Excess ACTH can cause the adrenal glands to produce too much cortisol. Over time, high cortisol levels can lead to a range of health problems, including metabolic and cardiovascular complications as well as neuropsychiatric effects. The disease can also increase the risk of serious complications and premature death.
Although existing medicines can help control excess cortisol, Lundbeck said there is still a significant need for better treatment options. Current therapies can have limitations related to effectiveness, safety and tolerability, making long-term disease control difficult for some patients.
Asedebart is being studied as a potential first-in-class treatment for diseases driven by excess ACTH. Lundbeck is currently conducting proof-of-concept clinical trials to assess the drug’s safety and effectiveness in Cushing’s disease and classic congenital adrenal hyperplasia (CAH).
The new European Union orphan designation adds to the regulatory recognition for asedebart. The drug has previously received orphan designations for CAH in the European Union and United States, as well as for CAH and Cushing’s disease in Japan.
Johan Luthman, Executive Vice President of Research and Development at Lundbeck, said the European designation highlights the unmet medical need in Cushing’s syndrome and supports the scientific approach behind asedebart.
The European Union’s orphan designation program is intended to encourage the development of medicines for rare, serious or chronically disabling diseases. Companies developing designated medicines can receive regulatory support and certain financial benefits, including fee reductions and protocol assistance.
If an orphan medicine is eventually approved, it may also qualify for market exclusivity for the designated indication in the European Union.
For Lundbeck, the designation strengthens the company’s growing pipeline of treatments targeting rare neuroendocrine disorders. The company plans to continue clinical development of asedebart as it evaluates its potential to provide a new treatment option for patients with ACTH-driven diseases.