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 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.
"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.
• Lu AG13909, an anti-ACTH antibody, is progressing into mid-stage clinical development as a novel therapy for conditions with elevated ACTH levels, including classic congenital adrenal hyperplasia (CAH).
• Lundbeck is expanding its ongoing Phase I/II open-label trial to further evaluate the efficacy and safety of Lu AG13909 in adults with classic CAH.
• The expanded trial will begin enrolment in North America and seven European countries, with the first clinical sites opening in late June 2025.
• This marks a significant step in developing a targeted, antibody-based treatment for CAH, a condition with limited therapeutic options.
• The expanded trial will enrol men and women, aged ≥18 to ≤70 years with classic CAH, under stable glucocorticoid dosing.
• Participants will receive monthly intravenous administrations of Lu AG13909 and will be divided into two cohorts.
• The first will include participants with hyperandrogenemia, and the second will include participants with normal androgen levels, but treated with supraphysiologic glucocorticoid doses.
• Participants may enter an optional open-label extension, where they receive monthly Lu AG13909 administration over a period of 12 months.
• Lu AG13909 is a humanised anti-ACTH monoclonal antibody that specifically recognises ACTH with high affinity.
• It blocks the binding of ACTH to the melanocortin 2 receptor in the adrenal glands and thereby inhibits the neurohormonal signalling of ACTH.
• This inhibition causes a decreased secretion of glucocorticoids, mineralocorticoids and androgens from the adrenal glands.
• ACTH plays a key role in the biosynthesis of adrenal steroids and is therefore considered a promising therapeutic target in conditions characterised by elevated ACTH levels.
• In this context, Lu AG13909, a novel molecule, may provide a therapeutic approach for treating conditions associated with chronically elevated ACTH levels.
In animal studies, Lu AG13909 has shown significant and durable reductions of corticosterone/cortisol and aldosterone.3 No adverse effects were observed after 6 months of intravenous dosing.
• Classic CAH is a rare, autosomal recessive disorder1 affecting 1 in 14,000–18,000 live births worldwide.
• Classic CAH is characterised by an enzyme deficiency, most commonly 21-hydroxylase deficiency, affecting the adrenal steroidogenesis leading to cortisol and aldosterone deficiency.
• People with 21-hydroxylase deficiency are at risk of adrenal crisis, a life-threatening condition contributing to the increased mortality throughout life.
• Balancing physiological glucocorticoid replacement and control of hyperandrogenism remains a challenge with the risk of long-term consequences of glucocorticoid overtreatment.
Contacts
Marie Petterson
Jens Høyer Palle Holm Olesen
Head of Media Relations, Corp. Communication
Vice President, Head of Investor Relations
MEEP@lundbeck.com
JSHR@lundbeck.com
+45 29 82 21 82
+45 30 83 45 01
Vice President, Investor Relations
PALO@lundbeck.com
+45 30 83 24 26
• Lundbeck is a biopharmaceutical company focusing exclusively on brain health.
• With more than 70 years of experience in neuroscience, we are committed to improving the lives of people with neurological and psychiatric diseases.
• About Brain disorders & H. Lundbeck A/S- Brain disorders affect a large part of the world's population, and the effects are felt throughout society.
• With the rapidly improving understanding of the biology of the brain, we hold ourselves accountable for advancing brain health by curiously exploring new opportunities for treatments.
• As a focused innovator, we strive for our research and development programs to tackle some of the most complex neurological challenges.
• We develop transformative medicines targeting people for whom there are few or no treatments available, expanding into neuro-specialty and neuro-rare from our strong legacy within psychiatry and neurology.
• We are committed to fighting stigma and we act to improve health equity.
• We strive to create long term value for our shareholders by making a positive contribution to patients, their families and society as a whole.
• Lundbeck has approximately 5,700 employees in more than 50 countries and our products are available in more than 80 countries.
• For additional information, we encourage you to visit our corporate site www.lundbeck.com and connect with us via LinkedIn.
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