Dyne Therapeutics' DYNE-101 Granted Orphan Drug Designation By EMA

(RTTNews) - Dyne Therapeutics, Inc. (DYN), a clinical-stage pharmaceutical company, Thursday, announced that its investigational drug DYNE-101 has been granted orphan drug designation by the European Medicines Agency.

The orphan drug designation is granted to drugs and biologics that treat, diagnose, or prevent rare or life-threatening diseases that affect fewer than 5 in ten thousand people in the European Union.

This designation gives certain benefits including reduced regulatory fees, clinical protocol assistance among others.

DYNE-101 is being evaluated in a phase I/II global clinical trial in adults with myotonic dystrophy type 1, dubbed ACHIEVE.

Myotonic Dystrophy Type 1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. More than 40,000 people in the United States and over 74,000 people in Europe are affected by this disease, for which there are no approved disease-modifying therapies.

Initial data from the multiple ascending dose portion of the ACHIEVE study is expected in the second half of 2023.

DYN is currently trading at $13.58, up 0.67%.