Dyne's muscle disorder therapy DYNE-101 gets orphan drug status in EU

May 25, 2023 9:47 AM ETDyne Therapeutics, Inc. (DYN)By: Ravikash, SA News Editor

The European Medicines Agency (EMA) granted orphan drug designation to Dyne Therapeutics' (NASDAQ:DYN) DYNE-101 to treat myotonic dystrophy type 1 (DM1).
DM1 is a disorder which affects muscles and several organs such as the eye, heart, endocrine system, and central nervous system.
The company noted that DYNE-101 is being evaluated in a phase 1/2 trial, dubbed ACHIEVE, in adults with DM1.
"The DM1 community has waited far too long for a therapy that addresses the underlying cause of this devastating rare muscle disease," said Dyne's Chief Medical Officer Wildon Farwell.
The orphan drug status in the EU is aimed at therapies treating or preventing diseases that affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years of market exclusivity for the drug, if approved.