{"success":true,"data":{"pressRelease":{"id":"141291","rtpr_id":"nPn9HD07ca-20260911","ticker":"HLUNA","exchange":"Nasdaq Copenhagen","all_tickers":["HLUNA"],"title":"The U.S. FDA grants orphan drug designation for Lundbeck's investigational anti-ACTH monoclonal antibody asedebart for treatment of endogenous Cushing's syndrome","author":"PR Newswire","published_at":"2026-09-11T08:22:15.609Z","article_body":"The U.S. FDA grants orphan drug designation for Lundbeck's investigational anti-ACTH monoclonal antibody asedebart for treatment of endogenous Cushing's syndrome\nPR Newswire\n\nVALBY, Denmark, Sept. 11, 2026\n\n * The U.S. FDA has granted Orphan Drug Designation for asedebart (Lu AG13909),\nLundbeck's novel investigational anti-ACTH monoclonal antibody, for the\ntreatment of endogenous Cushing's syndrome\n * Endogenous Cushing's Syndrome is a rare endocrine disorder driven in most\ncases by excess adrenocorticotropic hormone, or ACTH, leading to chronic\ncortisol excess and substantial disease burden, with current treatment options\noften limited by suboptimal disease control and treatment-related\ncomplications (1–3)\n * Proof-of-concept trial currently ongoing to evaluate efficacy and safety of Lu\nAG13909 in Cushing's disease (CD)(5)\nVALBY, Denmark, Sept. 11, 2026 /PRNewswire/ -- Lundbeck today announced that\nthe U.S. Food and Drug Administration (FDA) has granted Orphan Drug\nDesignation (ODD) to asedebart (Lu AG13909), Lundbeck's novel investigational\nanti-ACTH monoclonal antibody, for the treatment of endogenous Cushing's\nsyndrome.\n\nEndogenous Cushing's syndrome includes both ACTH-dependent and\nACTH-independent forms. ACTH-dependent Cushing's syndrome is a rare, serious\nendocrine disorder caused by excess secretion of adrenocorticotropic hormone\n(ACTH), most commonly from a pituitary tumor (Cushing's disease) and less\nfrequently from an ectopic ACTH-secreting tumor.(1) Elevated ACTH drives\nincreased adrenal production of glucocorticoids, mineralocorticoids and\nandrogens, disrupting normal physiological homeostasis. Of particular\nimportance is sustained cortisol excess, which contributes to a considerable\ndisease burden through metabolic, cardiovascular and neuropsychiatric\ncomplications and is associated with increased morbidity and mortality.(1,2)\nAlthough existing medical therapies can reduce or control elevated cortisol\nlevels, important treatment gaps remain.(1,3) Achieving and maintaining\nadequate disease control can be difficult, with available therapies differing\nin their efficacy and potentially constrained by safety and tolerability\nconsiderations.(4)\n\nAsedebart is a novel investigational monoclonal antibody targeting ACTH and is\nbeing developed for ACTH-dependent Cushing's syndrome. It is advancing in\nclinical development as a potential first-in-class treatment for rare\nconditions characterized by excess ACTH, with proof-of-concept trials ongoing\nin CD and classic congenital adrenal hyperplasia (CAH) to evaluate efficacy\nand safety.(5–8)\n\nWith this designation, asedebart continues to build momentum with regulatory\nauthorities across rare ACTH-driven disorders. Asedebart has also received\norphan designation in the European Union for Cushing's syndrome of endogenous\norigin, in addition to previous orphan drug designations for CAH in the\nEuropean Union and the United States, and for CAH and CD in Japan.(6,9,10)\n\n\"The FDA Orphan Drug Designation is an important step for asedebart and for\nLundbeck's growing commitment to rare neuroendocrine disorders,\" said Tarek\nSamad, Executive Vice President and Head of Research & Development at\nLundbeck. \"ACTH-dependent Cushing's syndrome can be a devastating condition\nfor patients, with long-term consequences that remain difficult to control\ndespite available treatments. This milestone reflects the strength of the\nscience behind asedebart's development to date and supports our ambition to\nadvance innovative medicines in areas where patients continue to face\nsignificant unmet need.\"\n\nOrphan Drug Designation is granted by the US FDA to drugs and biologics\nintended to treat, diagnose or prevent rare diseases or conditions. The\ndesignation may provide certain development incentives, including tax credits\nfor qualified clinical testing, exemption from certain FDA application fees\nand, if approved, potential seven years of market exclusivity for the\ndesignated indication.(11)\n\nAsedebart is an investigational compound that is not approved for marketing by\nany regulatory authority worldwide, and its efficacy and safety have not been\nestablished.\n\nAbout asedebart\n\nAsedebart is a humanized anti-ACTH monoclonal antibody that specifically\nrecognizes ACTH with high affinity. It blocks the binding of ACTH to the\nmelanocortin 2 receptor in the adrenal glands and thereby inhibits the\nneurohormonal signalling of ACTH. This inhibition reduces secretion of\nglucocorticoids, mineralocorticoids and androgens from the adrenal\nglands.(12,13)\n\nACTH plays a key role in the biosynthesis of adrenal steroids(13) and is\ntherefore considered a promising therapeutic target in conditions\ncharacterized by elevated ACTH levels. Through its mechanism of action,\nasedebart has the potential to treat conditions associated with chronically\nelevated ACTH levels, such as CD and CAH.\n\nAbout ACTH-dependent Cushing's syndrome\n\nACTH-dependent Cushing's syndrome is a rare, serious endocrine disorder caused\nby excess secretion of adrenocorticotropic hormone (ACTH), most commonly from\na pituitary tumor (Cushing's disease) and less frequently from an ectopic\nACTH-secreting tumor.(1) Chronic cortisol excess is associated with\nsubstantial disease burden, including metabolic, cardiovascular and\nneuropsychiatric complications, and is linked to increased morbidity and\nmortality.(1,2) Surgery to remove the source of excess ACTH is the preferred\nfirst-line treatment when feasible; however, not all patients are eligible for\nsurgery or achieve sustained remission. Current medical therapies can help\nmanage cortisol excess, but disease control remains challenging and treatment\noptions may be limited by variable efficacy, safety and tolerability.(1,3)\n\nContacts\n Anders Crillesen                                    Jens Høyer\n Senior Director, External & Internal Relations      Vice President, Head of Investor Relations\n AECE@lundbeck.com (mailto:AECE@lundbeck.com)        JSHR@lundbeck.com (mailto:JSHR@lundbeck.com)\n +45 27 79 12 86                                     +45 30 83 45 01\n\nAbout H. Lundbeck A/S\n\nLundbeck is a biopharmaceutical company focusing exclusively on brain health.\nWith more than 70 years of experience in neuroscience, we are committed to\nimproving the lives of people with neurological and psychiatric diseases.\n\nBrain disorders affect a large part of the world's population, and the effects\nare felt throughout society. With the rapidly improving understanding of the\nbiology of the brain, we hold ourselves accountable for advancing brain health\nby curiously exploring new opportunities for treatments.\n\nAs a focused innovator, we strive for our research and development programs to\ntackle some of the most complex neurological challenges. We develop\ntransformative medicines targeting people for whom there are few or no\ntreatments available, expanding into neuro-specialty and neuro-rare from our\nstrong legacy within psychiatry and neurology.\n\nWe are committed to fighting stigma and we act to improve health equity. We\nstrive to create long term value for our shareholders by making a positive\ncontribution to patients, their families and society as a whole.\n\nLundbeck has more than 5,000 employees in more than 20 countries and our\nproducts are available in more than 80 countries. For additional information,\nwe encourage you to visit our corporate site www.lundbeck.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=2351395690&u=https%3A%2F%2Fwww.lundbeck.com%2Fglobal&a=www.lundbeck.com)\nand connect with us via LinkedIn\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1257842558&u=https%3A%2F%2Fwww.linkedin.com%2Fcompany%2Flundbeck%2F&a=LinkedIn)\n.\n\nReferences:\n\n 1. Nieman LK. ACTH-Dependent Cushing's Syndrome: Pathophysiology. Endocr Rev.\n2022;43(2):117–171.\n 2. Fleseriu M, et al. Consensus on diagnosis and management of Cushing's disease.\nLancet Diabetes Endocrinol. 2021;9(12):847–875.\n 3. Nieman LK, et al. Treatment of Cushing's Syndrome: An Endocrine Society\nClinical Practice Guideline. J Clin Endocrinol Metab. 2015;100(8):2807–2831.\n 4. Julia Simões Corrêa Galendi\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=634582200&u=https%3A%2F%2Feur01.safelinks.protection.outlook.com%2F%3Furl%3Dhttps%253A%252F%252Fpubmed.ncbi.nlm.nih.gov%252F%253Fterm%253D%252522Sim%2525C3%2525B5es%252520Corr%2525C3%2525AAa%252520Galendi%252520J%252522%25255bAuthor%25255d%26data%3D05%257C02%257CCMFR%2540lundbeck.com%257Cf1fd52718f2049e7c0a208def9eab8bb%257Cca625151ac0044419024c88fad6084da%257C0%257C0%257C639222983680528605%257CUnknown%257CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%253D%253D%257C0%257C%257C%257C%26sdata%3DDryo%252FiPWB%252F%252FJfgPGv%252FXhSN05okQGBPvHiKM30XcPN24%253D%26reserved%3D0&a=Julia+Sim%C3%B5es+Corr%C3%AAa+Galendi)\n\n, et al. Effectiveness of Medical Treatment of Cushing's Disease: A\nSystematic Review and Meta-Analysis. Front Endocrinol 2021;12:732240.\n 5. ClinicalTrials.gov. A Trial of Lu AG13909 in Adult Participants With Cushing's\nDisease, BalanCeD. NCT06471829.\n 6. Lundbeck. Data on file.\n 7. Johannsson G, Velusamy A, Prete A, et al. MON-468: A Novel Anti-ACTH Antibody\nLu AG13909 in Clinical Development for Treatment of Classic Congenital Adrenal\nHyperplasia: A Phase 1 Open-Label, Multiple-Ascending-Dose Study. Journal of\nthe Endocrine Society. 2025;9(Suppl 1):bvaf149.226.\n 8. Srirangalingam U, Prete A, Frederiksen T, et al. SAT-455: A Phase 2,\nOpen-Label Trial Evaluating the Efficacy and Safety of Anti-ACTH Antibody Lu\nAG13909 in Adults With Classic Congenital Adrenal Hyperplasia: Trial Design.\nJournal of the Endocrine Society. 2025;9(Suppl 1):bvaf149.323.\n 9. Lundbeck. Lundbeck receives orphan drug designation in the US and EU for Lu\nAG13909 for the treatment of congenital adrenal hyperplasia. 24 June 2025.\n 10. Lundbeck. Lundbeck receives orphan drug designation in Japan for asedebart for\nthe treatment of patients with congenital adrenal hyperplasia and Cushing's\ndisease. 18 May 2026.\n 11. U.S. Food and Drug Administration. Designating an Orphan Product: Drugs and\nBiological Products.\n 12. Feldhaus AL, et al. ALD1613, a Novel Long-Acting Monoclonal Antibody to\nControl ACTH-Driven Pharmacology. Endocrinology. 2017;158(1):1–8.\n 13. Xing Y, et al. The effects of ACTH on steroid metabolomic profiles in human\nadrenal cells. J Endocrinol. 2011;209(3):327–335.\nCONTACT:\n\nH. Lundbeck A/S\nOttiliavej 9, 2500 Valby, Denmark\n+45 3630 1311\ninfo@lundbeck.com (mailto:info@lundbeck.com)\n\nThis information was brought to you by Cision http://news.cision.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1071153397&u=http%3A%2F%2Fnews.cision.com%2F&a=http%3A%2F%2Fnews.cision.com)\n\nhttps://news.cision.com/h--lundbeck-a-s/r/the-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti,c4394793\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1556264179&u=https%3A%2F%2Fnews.cision.com%2Fh--lundbeck-a-s%2Fr%2Fthe-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti%2Cc4394793&a=https%3A%2F%2Fnews.cision.com%2Fh--lundbeck-a-s%2Fr%2Fthe-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti%2Cc4394793)\n\nThe following files are available for download:\n https://mb.cision.com/Main/18215/4394793/4264047.pdf                                                                                                                                                                                   FDA_ODD_Cushings_Final\n (https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=995488375&u=https%3A%2F%2Fmb.cision.com%2FMain%2F18215%2F4394793%2F4264047.pdf&a=https%3A%2F%2Fmb.cision.com%2FMain%2F18215%2F4394793%2F4264047.pdf)\n\n \n\nView original\ncontent:https://www.prnewswire.com/news-releases/the-us-fda-grants-orphan-drug-designation-for-lundbecks-investigational-anti-acth-monoclonal-antibody-asedebart-for-treatment-of-endogenous-cushings-syndrome-302876245.html\n(https://www.prnewswire.com/news-releases/the-us-fda-grants-orphan-drug-designation-for-lundbecks-investigational-anti-acth-monoclonal-antibody-asedebart-for-treatment-of-endogenous-cushings-syndrome-302876245.html)\n\nSOURCE H. Lundbeck A/S\n\n\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved.","article_body_html":"","raw_payload":{"data":{"id":"nPn9HD07ca-20260911","title":"The U.S. FDA grants orphan drug designation for Lundbeck's investigational anti-ACTH monoclonal antibody asedebart for treatment of endogenous Cushing's syndrome","author":"PR Newswire","ticker":"HLUNA","created":"2026-09-11T08:22:15.609Z","tickers":["HLUNA"],"exchange":"Nasdaq Copenhagen","article_body":"The U.S. FDA grants orphan drug designation for Lundbeck's investigational anti-ACTH monoclonal antibody asedebart for treatment of endogenous Cushing's syndrome\nPR Newswire\n\nVALBY, Denmark, Sept. 11, 2026\n\n * The U.S. FDA has granted Orphan Drug Designation for asedebart (Lu AG13909),\nLundbeck's novel investigational anti-ACTH monoclonal antibody, for the\ntreatment of endogenous Cushing's syndrome\n * Endogenous Cushing's Syndrome is a rare endocrine disorder driven in most\ncases by excess adrenocorticotropic hormone, or ACTH, leading to chronic\ncortisol excess and substantial disease burden, with current treatment options\noften limited by suboptimal disease control and treatment-related\ncomplications (1–3)\n * Proof-of-concept trial currently ongoing to evaluate efficacy and safety of Lu\nAG13909 in Cushing's disease (CD)(5)\nVALBY, Denmark, Sept. 11, 2026 /PRNewswire/ -- Lundbeck today announced that\nthe U.S. Food and Drug Administration (FDA) has granted Orphan Drug\nDesignation (ODD) to asedebart (Lu AG13909), Lundbeck's novel investigational\nanti-ACTH monoclonal antibody, for the treatment of endogenous Cushing's\nsyndrome.\n\nEndogenous Cushing's syndrome includes both ACTH-dependent and\nACTH-independent forms. ACTH-dependent Cushing's syndrome is a rare, serious\nendocrine disorder caused by excess secretion of adrenocorticotropic hormone\n(ACTH), most commonly from a pituitary tumor (Cushing's disease) and less\nfrequently from an ectopic ACTH-secreting tumor.(1) Elevated ACTH drives\nincreased adrenal production of glucocorticoids, mineralocorticoids and\nandrogens, disrupting normal physiological homeostasis. Of particular\nimportance is sustained cortisol excess, which contributes to a considerable\ndisease burden through metabolic, cardiovascular and neuropsychiatric\ncomplications and is associated with increased morbidity and mortality.(1,2)\nAlthough existing medical therapies can reduce or control elevated cortisol\nlevels, important treatment gaps remain.(1,3) Achieving and maintaining\nadequate disease control can be difficult, with available therapies differing\nin their efficacy and potentially constrained by safety and tolerability\nconsiderations.(4)\n\nAsedebart is a novel investigational monoclonal antibody targeting ACTH and is\nbeing developed for ACTH-dependent Cushing's syndrome. It is advancing in\nclinical development as a potential first-in-class treatment for rare\nconditions characterized by excess ACTH, with proof-of-concept trials ongoing\nin CD and classic congenital adrenal hyperplasia (CAH) to evaluate efficacy\nand safety.(5–8)\n\nWith this designation, asedebart continues to build momentum with regulatory\nauthorities across rare ACTH-driven disorders. Asedebart has also received\norphan designation in the European Union for Cushing's syndrome of endogenous\norigin, in addition to previous orphan drug designations for CAH in the\nEuropean Union and the United States, and for CAH and CD in Japan.(6,9,10)\n\n\"The FDA Orphan Drug Designation is an important step for asedebart and for\nLundbeck's growing commitment to rare neuroendocrine disorders,\" said Tarek\nSamad, Executive Vice President and Head of Research & Development at\nLundbeck. \"ACTH-dependent Cushing's syndrome can be a devastating condition\nfor patients, with long-term consequences that remain difficult to control\ndespite available treatments. This milestone reflects the strength of the\nscience behind asedebart's development to date and supports our ambition to\nadvance innovative medicines in areas where patients continue to face\nsignificant unmet need.\"\n\nOrphan Drug Designation is granted by the US FDA to drugs and biologics\nintended to treat, diagnose or prevent rare diseases or conditions. The\ndesignation may provide certain development incentives, including tax credits\nfor qualified clinical testing, exemption from certain FDA application fees\nand, if approved, potential seven years of market exclusivity for the\ndesignated indication.(11)\n\nAsedebart is an investigational compound that is not approved for marketing by\nany regulatory authority worldwide, and its efficacy and safety have not been\nestablished.\n\nAbout asedebart\n\nAsedebart is a humanized anti-ACTH monoclonal antibody that specifically\nrecognizes ACTH with high affinity. It blocks the binding of ACTH to the\nmelanocortin 2 receptor in the adrenal glands and thereby inhibits the\nneurohormonal signalling of ACTH. This inhibition reduces secretion of\nglucocorticoids, mineralocorticoids and androgens from the adrenal\nglands.(12,13)\n\nACTH plays a key role in the biosynthesis of adrenal steroids(13) and is\ntherefore considered a promising therapeutic target in conditions\ncharacterized by elevated ACTH levels. Through its mechanism of action,\nasedebart has the potential to treat conditions associated with chronically\nelevated ACTH levels, such as CD and CAH.\n\nAbout ACTH-dependent Cushing's syndrome\n\nACTH-dependent Cushing's syndrome is a rare, serious endocrine disorder caused\nby excess secretion of adrenocorticotropic hormone (ACTH), most commonly from\na pituitary tumor (Cushing's disease) and less frequently from an ectopic\nACTH-secreting tumor.(1) Chronic cortisol excess is associated with\nsubstantial disease burden, including metabolic, cardiovascular and\nneuropsychiatric complications, and is linked to increased morbidity and\nmortality.(1,2) Surgery to remove the source of excess ACTH is the preferred\nfirst-line treatment when feasible; however, not all patients are eligible for\nsurgery or achieve sustained remission. Current medical therapies can help\nmanage cortisol excess, but disease control remains challenging and treatment\noptions may be limited by variable efficacy, safety and tolerability.(1,3)\n\nContacts\n Anders Crillesen                                    Jens Høyer\n Senior Director, External & Internal Relations      Vice President, Head of Investor Relations\n AECE@lundbeck.com (mailto:AECE@lundbeck.com)        JSHR@lundbeck.com (mailto:JSHR@lundbeck.com)\n +45 27 79 12 86                                     +45 30 83 45 01\n\nAbout H. Lundbeck A/S\n\nLundbeck is a biopharmaceutical company focusing exclusively on brain health.\nWith more than 70 years of experience in neuroscience, we are committed to\nimproving the lives of people with neurological and psychiatric diseases.\n\nBrain disorders affect a large part of the world's population, and the effects\nare felt throughout society. With the rapidly improving understanding of the\nbiology of the brain, we hold ourselves accountable for advancing brain health\nby curiously exploring new opportunities for treatments.\n\nAs a focused innovator, we strive for our research and development programs to\ntackle some of the most complex neurological challenges. We develop\ntransformative medicines targeting people for whom there are few or no\ntreatments available, expanding into neuro-specialty and neuro-rare from our\nstrong legacy within psychiatry and neurology.\n\nWe are committed to fighting stigma and we act to improve health equity. We\nstrive to create long term value for our shareholders by making a positive\ncontribution to patients, their families and society as a whole.\n\nLundbeck has more than 5,000 employees in more than 20 countries and our\nproducts are available in more than 80 countries. For additional information,\nwe encourage you to visit our corporate site www.lundbeck.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=2351395690&u=https%3A%2F%2Fwww.lundbeck.com%2Fglobal&a=www.lundbeck.com)\nand connect with us via LinkedIn\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1257842558&u=https%3A%2F%2Fwww.linkedin.com%2Fcompany%2Flundbeck%2F&a=LinkedIn)\n.\n\nReferences:\n\n 1. Nieman LK. ACTH-Dependent Cushing's Syndrome: Pathophysiology. Endocr Rev.\n2022;43(2):117–171.\n 2. Fleseriu M, et al. Consensus on diagnosis and management of Cushing's disease.\nLancet Diabetes Endocrinol. 2021;9(12):847–875.\n 3. Nieman LK, et al. Treatment of Cushing's Syndrome: An Endocrine Society\nClinical Practice Guideline. J Clin Endocrinol Metab. 2015;100(8):2807–2831.\n 4. Julia Simões Corrêa Galendi\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=634582200&u=https%3A%2F%2Feur01.safelinks.protection.outlook.com%2F%3Furl%3Dhttps%253A%252F%252Fpubmed.ncbi.nlm.nih.gov%252F%253Fterm%253D%252522Sim%2525C3%2525B5es%252520Corr%2525C3%2525AAa%252520Galendi%252520J%252522%25255bAuthor%25255d%26data%3D05%257C02%257CCMFR%2540lundbeck.com%257Cf1fd52718f2049e7c0a208def9eab8bb%257Cca625151ac0044419024c88fad6084da%257C0%257C0%257C639222983680528605%257CUnknown%257CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%253D%253D%257C0%257C%257C%257C%26sdata%3DDryo%252FiPWB%252F%252FJfgPGv%252FXhSN05okQGBPvHiKM30XcPN24%253D%26reserved%3D0&a=Julia+Sim%C3%B5es+Corr%C3%AAa+Galendi)\n\n, et al. Effectiveness of Medical Treatment of Cushing's Disease: A\nSystematic Review and Meta-Analysis. Front Endocrinol 2021;12:732240.\n 5. ClinicalTrials.gov. A Trial of Lu AG13909 in Adult Participants With Cushing's\nDisease, BalanCeD. NCT06471829.\n 6. Lundbeck. Data on file.\n 7. Johannsson G, Velusamy A, Prete A, et al. MON-468: A Novel Anti-ACTH Antibody\nLu AG13909 in Clinical Development for Treatment of Classic Congenital Adrenal\nHyperplasia: A Phase 1 Open-Label, Multiple-Ascending-Dose Study. Journal of\nthe Endocrine Society. 2025;9(Suppl 1):bvaf149.226.\n 8. Srirangalingam U, Prete A, Frederiksen T, et al. SAT-455: A Phase 2,\nOpen-Label Trial Evaluating the Efficacy and Safety of Anti-ACTH Antibody Lu\nAG13909 in Adults With Classic Congenital Adrenal Hyperplasia: Trial Design.\nJournal of the Endocrine Society. 2025;9(Suppl 1):bvaf149.323.\n 9. Lundbeck. Lundbeck receives orphan drug designation in the US and EU for Lu\nAG13909 for the treatment of congenital adrenal hyperplasia. 24 June 2025.\n 10. Lundbeck. Lundbeck receives orphan drug designation in Japan for asedebart for\nthe treatment of patients with congenital adrenal hyperplasia and Cushing's\ndisease. 18 May 2026.\n 11. U.S. Food and Drug Administration. Designating an Orphan Product: Drugs and\nBiological Products.\n 12. Feldhaus AL, et al. ALD1613, a Novel Long-Acting Monoclonal Antibody to\nControl ACTH-Driven Pharmacology. Endocrinology. 2017;158(1):1–8.\n 13. Xing Y, et al. The effects of ACTH on steroid metabolomic profiles in human\nadrenal cells. J Endocrinol. 2011;209(3):327–335.\nCONTACT:\n\nH. Lundbeck A/S\nOttiliavej 9, 2500 Valby, Denmark\n+45 3630 1311\ninfo@lundbeck.com (mailto:info@lundbeck.com)\n\nThis information was brought to you by Cision http://news.cision.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1071153397&u=http%3A%2F%2Fnews.cision.com%2F&a=http%3A%2F%2Fnews.cision.com)\n\nhttps://news.cision.com/h--lundbeck-a-s/r/the-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti,c4394793\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=1556264179&u=https%3A%2F%2Fnews.cision.com%2Fh--lundbeck-a-s%2Fr%2Fthe-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti%2Cc4394793&a=https%3A%2F%2Fnews.cision.com%2Fh--lundbeck-a-s%2Fr%2Fthe-u-s--fda-grants-orphan-drug-designation-for-lundbeck-s-investigational-anti-acth-monoclonal-anti%2Cc4394793)\n\nThe following files are available for download:\n https://mb.cision.com/Main/18215/4394793/4264047.pdf                                                                                                                                                                                   FDA_ODD_Cushings_Final\n (https://edge.prnewswire.com/c/link/?t=0&l=en&o=4772127-1&h=995488375&u=https%3A%2F%2Fmb.cision.com%2FMain%2F18215%2F4394793%2F4264047.pdf&a=https%3A%2F%2Fmb.cision.com%2FMain%2F18215%2F4394793%2F4264047.pdf)\n\n \n\nView original\ncontent:https://www.prnewswire.com/news-releases/the-us-fda-grants-orphan-drug-designation-for-lundbecks-investigational-anti-acth-monoclonal-antibody-asedebart-for-treatment-of-endogenous-cushings-syndrome-302876245.html\n(https://www.prnewswire.com/news-releases/the-us-fda-grants-orphan-drug-designation-for-lundbecks-investigational-anti-acth-monoclonal-antibody-asedebart-for-treatment-of-endogenous-cushings-syndrome-302876245.html)\n\nSOURCE H. Lundbeck A/S\n\n\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved."},"type":"article","timestamp":"2026-09-11T08:22:15.662628919Z","server_sent_at_ms":1789114935662},"received_at":"2026-09-11T08:22:15.717Z","source_url":"https://www.prnewswire.com/news-releases/the-us-fda-grants-orphan-drug-designation-for-lundbecks-investigational-anti-acth-monoclonal-antibody-asedebart-for-treatment-of-endogenous-cushings-syndrome-302876245.html"},"analysis":{"id":"130127","press_release_id":"141291","analysis_json":{"industry":{"label":"Pharmaceuticals","sector":"Health Care"},"redFlags":["asedebart is investigational and not approved by any regulatory authority; efficacy and safety not established","orphan drug designation confers incentives only — approval, PoC data readouts, and commercial timelines remain ahead"],"eventType":"regulatory","narrative":"Lundbeck received U.S. FDA Orphan Drug Designation for asedebart (Lu AG13909), its investigational anti-ACTH monoclonal antibody, as a treatment for endogenous Cushing's syndrome, a rare endocrine disorder driven by chronic cortisol excess.\n\nThe designation provides development incentives including tax credits for qualified clinical testing, FDA application fee exemptions, and, if approved, potential seven years of U.S. market exclusivity. Asedebart already holds orphan designations in the EU for Cushing's syndrome, in the US and EU for congenital adrenal hyperplasia, and in Japan for both CAH and Cushing's disease.\n\nThe asset remains investigational with no marketing approval anywhere and unestablished efficacy and safety; proof-of-concept trials are ongoing in Cushing's disease and classic congenital adrenal hyperplasia. This is a pipeline-momentum signal for Lundbeck's rare-disease franchise rather than a near-term revenue event.","sentiment":"bullish","agentHooks":{"shouldPost":false,"suggestedAngle":"FDA orphan designation extends asedebart's regulatory momentum in rare ACTH-driven disorders, but approval and revenue remain years away."},"keyFigures":{"drugName":"asedebart (Lu AG13909)","phaseOfTrial":"Proof-of-concept","customDimensions":{"potential_market_exclusivity_years_us_if_approved":7}},"quotedText":"ACTH-dependent Cushing's syndrome can be a devastating condition","namedEntities":{"people":[{"name":"Tarek Samad","role":"Executive Vice President and Head of Research & Development, Lundbeck"}],"products":["asedebart","Lu AG13909"],"companies":[{"name":"H. 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It grants development incentives (tax credits, fee exemptions, potential 7-year exclusivity if approved) but has no near-term revenue or binary-outcome impact."},"tickerRelevance":{"others":[],"primary":"HLUNA"},"globalImportance":22,"audienceRelevance":20,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"mid-cap European pharma with thinly traded US ADR","eventGravity":"routine early-stage regulatory designation (ODD)","sectorWeight":"pharma pipeline milestone","issuerAuthored":true,"householdBrandBoost":0,"retailFavoriteBoost":0}},"event_type":"regulatory","event_type_secondary":null,"sentiment":"bullish","material_impact_score":2,"narrative":"Lundbeck received U.S. FDA Orphan Drug Designation for asedebart (Lu AG13909), its investigational anti-ACTH monoclonal antibody, as a treatment for endogenous Cushing's syndrome, a rare endocrine disorder driven by chronic cortisol excess.\n\nThe designation provides development incentives including tax credits for qualified clinical testing, FDA application fee exemptions, and, if approved, potential seven years of U.S. market exclusivity. Asedebart already holds orphan designations in the EU for Cushing's syndrome, in the US and EU for congenital adrenal hyperplasia, and in Japan for both CAH and Cushing's disease.\n\nThe asset remains investigational with no marketing approval anywhere and unestablished efficacy and safety; proof-of-concept trials are ongoing in Cushing's disease and classic congenital adrenal hyperplasia. This is a pipeline-momentum signal for Lundbeck's rare-disease franchise rather than a near-term revenue event.","key_figures":{"drugName":"asedebart (Lu AG13909)","phaseOfTrial":"Proof-of-concept","customDimensions":{"potential_market_exclusivity_years_us_if_approved":7}},"named_entities":{"people":[{"name":"Tarek Samad","role":"Executive Vice President and Head of Research & Development, Lundbeck"}],"products":["asedebart","Lu AG13909"],"companies":[{"name":"H. Lundbeck A/S","ticker":"HLUNA","relationship":"filer"},{"name":"U.S. Food and Drug Administration","relationship":"regulatory authority"}],"dollarAmounts":[]},"model_name":"glm-5.3-flash","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-11T08:22:57.650Z","global_importance":22,"audience_relevance":20,"importance_components":{"tickerTier":"mid-cap European pharma with thinly traded US ADR","eventGravity":"routine early-stage regulatory designation (ODD)","sectorWeight":"pharma pipeline milestone","issuerAuthored":true,"householdBrandBoost":0,"retailFavoriteBoost":0}},"durationMs":41924,"modelName":"glm-5.3-flash"}}