DYNE-251 for DMD Granted FDA Breakthrough Therapy Designation

DYNE-251 for DMD granted FDA breakthrough therapy designation

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation is intended to accelerate the development and regulatory review of medications intended to treat serious conditions. Benefits include enhanced FDA support in development and decision-making, regular communication wi…

Dyne Therapeutics granted Breakthrough Therapy Designation for exon-51skipping therapeutic, DYNE-251

CureDuchenne was an early investor in Dyne Therapeutics in 2020, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for DYNE-251, their exon 51-skipping agent for the treatment of Duchenne. Why is this important: The FDA grants Breakthrough Therapy Designations to speed up the development and review of drugs for serious conditions when preliminary clinical evidence indicates that the drug may demonstrate…