Orphan designation: RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5') (C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-[2'a-[39-[[1-acetyl-L-prolyl-L-lysyl-L-lysyl-L-lysyl-L-arginyl-L-lysyl-L-valyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-(L-phenylalanylglycyl-L-phenylalanylglycyl-L-arginylglycyl-L-arginyl-L-gamma-glutamyl)-L-lysyl]amino]-1- oxo-4,7,10,13,16,19,22,25,28,31,34,37-dodecaoxanonatriacont-1-yl]]G), (8'->1')-lactam Treatment of dystrophic myotonia, 22/08/2025 Positive

EU/3/25/3105 - orphan designation for treatment of dystrophic myotonia RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5') (C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-[2'a-[39-[[1-acetyl-L-prolyl-L-lysyl-L-lysyl-L-lysyl-L-arginyl-L-lysyl-L-valyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-(L-phenylalanylglycyl-L-phenylalanylglycyl-L-arginylglycyl-L-arginyl-L-gamma-glutamyl)-L-lysyl]amino]-1- oxo-4,7,10,13,16,19,22,25,28,31,34,37-dodecaoxanonatriacont-1-yl]]G), (8'->1')-lactam Orphan Human Overview This medicine was designated as an orphan medicine for the treatment of dystrophic myotonia in the European Union on 22 August 2025. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation. Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU. During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see: EU Clinical Trials Register ClinicalTrials.gov Key facts Active substance RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5') (C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-[2'a-[39-[[1-acetyl-L-prolyl-L-lysyl-L-lysyl-L-lysyl-L-arginyl-L-lysyl-L-valyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-(L-phenylalanylglycyl-L-phenylalanylglycyl-L-arginylglycyl-L-arginyl-L-gamma-glutamyl)-L-lysyl]amino]-1- oxo-4,7,10,13,16,19,22,25,28,31,34,37-dodecaoxanonatriacont-1-yl]]G), (8'->1')-lactam Intended use Treatment of dystrophic myotonia Orphan designation status Positive EU designation number EU/3/25/3105 Date of designation 22/08/2025 Sponsor Vertex Pharmaceuticals (Ireland) Limited EMA list of opinions on orphan medicinal product designation EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform: List of opinions on orphan medicinal product designation Patients' organisations For contact details of patients’ organisations whose activities are targeted at rare diseases, see: European Organisation for Rare Diseases (EURORDIS) , a non-governmental alliance of patient organisations and individuals active in the field of rare diseases. Orphanet , a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe. EU register of orphan medicines The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: European Commission: Community Register of orphan medicinal products Share this page