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

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