FDA grants fast-track designation to myotonic dystrophy type 1 treatment

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic dystrophy type 1, or DM1, according to the manufacturer.

In a press release, Dyne Therapeutics Inc. stated DYNE-101, which was developed as a therapeutic that targets muscle to reduce harmful RNA in a cell nucleus with the goal of either slowing or arresting disease progression, is currently being evaluated in the ongoing phase 1/2 ACHIEVE global clinical trial.

DYNE-101 had previously been granted orphan drug designation by both the FDA and the European Medicines Agency for the treatment of DM1, a monogenic, autosomal dominant disease that causes muscle weakness throughout the body and is estimated to affect almost 115,000 people in the United States and Europe.

“DM1 is a devastating disease with no approved therapies, and we are driven to deliver DYNE-101, a potentially transformative medicine, to patients as quickly as possible,” Doug Kerr, MD, PhD, chief medical officer of Dyne, said in the release.