FDA grants fast track designation to myotonic dystrophy drug

The FDA has granted fast track designation to an investigational peptide-conjugated antisense oligonucleotide delivery system to treat myotonic dystrophy type 1, according to the manufacturer.

According to a press release from PepGen Inc., its novel therapeutic PGN-EDODM1 is currently being evaluated in the ongoing phase 1 FREEDOM-DM1 clinical trial, for which the company expects to report initial data later this year.

Fast track designation aims to facilitate development and expedite the review of drug candidates that treat serious conditions and fill unmet or urgent medical needs, ultimately paving the way for these therapeutic candidates to reach the patient earlier.

The FDA had previously granted PGN-EDODM-1 orphan drug designation in September 2023.

“Patients with DM1 currently have no available treatment options that target the root cause of the disease, which leads to progressive neuromuscular symptoms and reduction in life expectancy,” PepGen President and CEO James McArthur, PhD, said in the release. “We believe that PGN-EDODM1 has the potential to be disease-modifying and improve outcomes for patients living with [myotonic dystrophy type 1].”