Experimental Cushing’s treatment scores orphan drug status in Europe
Proof-of-concept trial in US, EU now testing therapy candidate
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Asedebart, an experimental treatment designed to ease symptoms in Cushing’s disease and other forms of endogenous Cushing’s syndrome, has been granted orphan drug status in the European Union.
That designation, awarded to the Lundbeck therapy by the European Commission, aims to incentivize the development of treatments for rare health conditions, specifically those affecting fewer than 5 of every 10,000 people across the EU. Such status will provide the developer with perks that include reduced fees and greater regulatory access, as well as a guaranteed decade of market exclusivity in the region if asedebart is ultimately approved. Asedebart has previously received a similar designation in Japan.
“Orphan designation in the European Union is an important recognition of both the unmet need in Cushing’s and the scientific rationale behind asedebart,” Johan Luthman, executive vice president of research and development at Lundbeck, said in a company press release.
Luthman added that this designation is “another example of a breakthrough innovation pipeline in neuroendocrine [hormone-related] and rare disorders, where several programs have been granted a number of special regulatory designations in recent years.”
Cushing’s syndrome is a broad term for health conditions driven by abnormally high levels of the hormone cortisol. In endogenous Cushing’s syndrome, high cortisol levels are caused by problems within the body itself, usually a tumor.
Cushing’s disease is the most common form of endogenous Cushing’s syndrome. It is caused by a tumor that forms in the brain’s pituitary gland and that secretes excessive amounts of adrenocorticotropic hormone (ACTH). This signaling molecule triggers the adrenal glands, located above the kidneys, to make cortisol, often called the stress hormone.
Elevated levels of cortisol ultimately drive Cushing’s symptoms such as weight changes, skin abnormalities, and mental health issues.
Trial testing asedebart now recruiting in 9 countries
Asedebart is an antibody-based therapy designed to block the activity of ACTH, thereby limiting cortisol production and ultimately easing symptoms.
Lundbeck is now running a small proof-of-concept clinical trial to test the effects of asedebart in adults with a confirmed diagnosis of Cushing’s disease. The Phase 2 study, called BalanCeD (NCT06471829), is still recruiting an estimated 18 participants at more than two dozen sites across the U.S. and Europe in nine countries. Eligible participants will not have any major co-occurring health issues and must be age 70 or younger.
All participants will be treated with asedebart, given via infusion into the bloodstream or injection under the skin, with the main goal of tracking the medications’ effect on cortisol levels.
Early data from the trial suggest that asedebart is generally tolerated well and reduces cortisol levels as designed. The study is expected to end in 2027.
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