Lundbeck has announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to asedebart (Lu AG13909) for the treatment of Congenital Adrenal Hyperplasia (CAH) and Cushing’s Disease (CD).

The designation supports the development of asedebart, a novel monoclonal antibody targeting Adrenocorticotropic Hormone (ACTH), as a potential first-in-class therapy for disorders linked to excessive ACTH production.

CAH and Cushing’s disease are rare endocrine disorders with different causes but a common underlying mechanism involving elevated ACTH levels. In CAH, impaired cortisol production leads to chronically high ACTH levels, resulting in excessive adrenal androgen production and the need for lifelong hormone replacement therapy. Cushing’s disease, on the other hand, is caused by overproduction of ACTH from a pituitary adenoma, leading to chronic cortisol excess.

According to the company, CAH affects approximately one in 14,000 to 18,000 live births globally, while Cushing’s disease has an estimated prevalence of around 2.2 cases per 100,000 people worldwide. Both disorders are associated with significant metabolic, cardiovascular and neuropsychiatric complications and are linked to increased morbidity and mortality.

Lundbeck stated that current treatment approaches for these conditions are aimed at managing hormonal imbalance but often face limitations related to efficacy and tolerability. By directly targeting ACTH, asedebart is intended to address the underlying disease biology rather than only managing symptoms.

Johan Luthman, Executive Vice President and Head of Research and Development at Lundbeck, said the designation reflects the company’s commitment to expanding its work in rare diseases and endocrinological conditions linked to brain function. He added that the recognition in Japan is an important step toward addressing unmet medical needs for patients living with CAH and Cushing’s disease.

Asedebart has already received orphan drug designation for CAH in both the European Union and the United States. Lundbeck is currently conducting proof-of-concept clinical trials to evaluate the safety and efficacy of the therapy in patients with classic CAH and Cushing’s disease.

The company noted that asedebart remains an investigational therapy and has not yet been approved for commercial use by any regulatory authority worldwide.