Orphan drug status for treatment of myotonic dystrophy type 1

Orphan drug status for treatment of myotonic dystrophy type 1  

With no cures for myotonic dystrophy type 1 and very few treatment options available, we’re pleased to see a new drug in clinical development. Yesterday Avidity Biosciences announced that the European Medicine Agency (EMA), the drugs regulatory authority for Europe, has granted their drug, AOC 1001, orphan designation

AOC 1001 is an experimental drug that targets the underlying cause of myotonic dystrophy type 1, a progressive, muscle-wasting condition that affects about 1 in 8,000 people in the UK (although some estimates suggest that this number could be higher). Orphan designation encourages pharmaceutical companies to invest in the development of drugs for rare conditions (those affecting fewer than five in 10,000 people). 

Earlier this summer, the Food and Drug Administration in the USA granted orphan designation to AOC 1001 and Avidity Biosciences hopes to start phase 1/2 clinical trials there in the near future. We will provide updates with more news about trials of AOC 1001, when we learn more. 

For more information, read Avidity Bioscience’s press release

If you have any research questions, please contact our Research Line on research@musculardystrophyuk.org