Asedebart for Cushing’s disease granted orphan drug status in Japan
Asian nation is one of developer Lundbeck 'focus markets' for new therapies
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Japan’s Ministry of Health, Labour and Welfare has granted orphan drug designation to Lundbeck’s asedebart, an investigational therapy for Cushing’s disease and congenital adrenal hyperplasia (CAH), two conditions characterized by excessive production of adrenocorticotropic hormone.
This regulatory status is intended to support the development of medicines for serious rare diseases with high unmet medical needs. In Japan, orphan drug designation may be granted for therapies targeting diseases affecting fewer than 50,000 people or certain intractable diseases, and can provide benefits such as regulatory consultation, priority review, fee reductions, and other development incentives.
“The investigational program for asedebart demonstrates Lundbeck’s commitment to deepen our efforts in rare diseases and endocrinological conditions with links to brain function,” Johan Luthman, executive vice president and head of research and development at Lundbeck, said in a company press release announcing the decision, noting that this award “also illustrates our approach to directly target underlying disease biology.”
Luthman noted that the Asian nation is one of the “focus markets” for Lundbeck’s drug development efforts.
“We are especially pleased to receive [orphan drug designation] for asedebart in Japan and the opportunity this provides to address significant unmet needs for people living with CAH and [Cushing’s disease],” Luthman said.
Cushing’s disease is caused by a tumor in the pituitary gland, a small organ at the base of the brain. The tumor releases too much adrenocorticotropic hormone (ACTH), which signals the adrenal glands to produce cortisol. Over time, excess cortisol can cause a wide range of symptoms and complications.
Proof-of-concept trials now testing asedebart’s safety, effectiveness
First-line treatment for Cushing’s disease is surgery to remove the pituitary tumor. However, not all patients are eligible for surgery, and some do not achieve lasting remission.
According to Lundbeck, existing medical treatments can have variable effectiveness and may be limited by safety or tolerability issues.
CAH has a different cause, an enzyme deficiency, but also involves abnormally high ACTH levels. In classic CAH, impaired cortisol production leads to chronically elevated ACTH, which can drive excess production of adrenal androgens, a group of sex hormones. People with CAH generally require lifelong hormone replacement therapy.
Asedebart, formerly known as Lu AG13909, is a humanized antibody that recognizes ACTH and blocks its signaling at the adrenal glands. By interfering with ACTH signaling, the therapy is expected to reduce the adrenal gland’s production of steroid hormones, including cortisol.
Lundbeck stated that it is conducting proof-of-concept clinical trials to evaluate asedebart’s safety and early effectiveness in people with classic CAH and Cushing’s disease.
