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Lundbeck receives orphan drug designation in the US and EU for Lu AG13909 for the treatment of patients with congenital adrenal hyperplasia
Lundbeck today announces that orphan drug designation has been granted to Lu AG13909 by the US Food and Drug Administration (FDA) on 12 May 2025 and the European Medicines Agency (EMA) on 20 June 2025. Lu AG13909 is a novel, humanised monoclonal antibody, under investigation for the treatment of patients with CAH, a rare genetic disease. CAH is characterised by impaired cortisol production and elevated levels of adrenocorticotropic hormone (ACTH), a hormone produced in the brain and involved in
About this update from H. Lundbeck A/s Class A
[{"type":"list","items":[{"val":[{"type":"text","content":"Congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disease1 with an estimated global incidence of approximately 1 in 14,000–18,000 live births2","length":159,"tagName":"p"}]},{"val":[{"type":"text","content":"A phase II trial has recently been initiated to investigate the efficacy and safety of LuAG13909 for the potential treatment of CAH3","length":132,"tagName":"p"}]}],"tagName":"ul","bulletedList":true,"length":291,"olType":false},{"type":"text","content":"VALBY, Denmark, June 24, 2025 /PRNewswire/ -- Lundbeck today announces that orphan drug designation has been granted to Lu AG13909 by the US Food and Drug Administration (FDA) on 12 May 2025 and the European Medicines Agency (EMA) on 20 June 2025. Lu AG13909 is a novel, humanised monoclonal antibody, under investigation for the treatment of patients with CAH, a rare genetic disease. CAH is characterised by impaired cortisol production and elevated levels of adrenocorticotropic hormone (ACTH), a hormone produced in the brain and involved in the regulation of multiple functions in the body. Elevated levels of ACTH lead to additional adrenal hormone imbalance, ultimately resulting in multiple developmental disturbances including symptoms related to the central nervous system, and long-term health concerns.","length":819,"tagName":"p"},{"type":"text","content":""CAH is a life-long condition, requiring constant management. Many existing treatments focus on controlling cortisol levels, however these options are often complicated by side effects. The orphan drug designation for Lu AG13909, our potential first in class anti-ACTH antibody, reflects the program's innovative approach, as well as the high medical need to find new treatments for CAH," said Johan Luthman, EVP and Head of Research and Development at Lundbeck.","length":476,"tagName":"p"},{"type":"text","content":"Lu AG13909 is advancing to mid-stage clinical development, as a new therapeutic approach for conditions marked by elevated ACTH levels, such as CAH. Lundbeck is currently expanding an ongoing Phase I/II clinical open-label trial, evaluating the efficacy and safety of anti-ACTH antibody Lu AG13909 in adults with classic CAH. The trial will open for enrolment in North America and seven countries across Europe, with the first sites opening in late Ju...