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In the European Union (EU), the European Medicines Agency (EMA) defines a drug as "orphan" if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically and seriously debilitating condition affecting not more than 5 in 10,000 EU people. [6]
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency ( EMEA ).
In July 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency recommended a conditional marketing authorization for epcoritamab (Tepkinly). [14] It was approved for medical use in the European Union in September 2023. [5] The EMA granted orphan drug designation to epcoritamab in both February and June 2022 ...
Pasireotide, sold under the brand name Signifor, is an orphan drug approved in the United States [1] and the European Union [2] [3] for the treatment of Cushing's disease in patients who fail or are ineligible for surgical therapy. [4] [5] [6] It was developed by Novartis.
Orphanet is an organisation and knowledge base dedicated to rare diseases as well as corresponding diagnosis, orphan drugs, clinical trials and expert networks. [1] Orphanet was founded in France in 1997 by Inserm, the French National Institute of Health and Medical Research. The website is managed by a network of academic establishments from ...
European Union, UK, US, India, South Africa, others Cardiac valvular disease, pulmonary hypertension, cardiac fibrosis; [ 3 ] [ 23 ] re-approved in June 2020 for the treatment of seizures associated with Dravet syndrome , under FDA orphan drug rules.
In October 2014, an orphan designation was granted by the European Commission for osilodrostat for the treatment of Cushing's syndrome. [9]Osilodrostat was approved for medical use in the European Union in January 2020, [5] and for medical use in the United States in March 2020.
The FDA classified odevixibat as an orphan drug for the rare conditions of Alagille syndrome, biliary atresia, and primary biliary cholangitis. [21] Odevixibat was granted its initial approval in July 2021, in the European Union for the treatment of progressive familial intrahepatic cholestasis in people aged six months and older.