Dyne Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.
“This Fast Track designation comes on the heels of robust clinical data from our ACHIEVE trial, which demonstrated substantial functional benefit for patients across a range of clinical measures and a compelling effect on the key disease biomarker of splicing correction,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “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.”
The FDA grants Fast Track designation to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. Rolling review allows a company to submit completed sections of its Biologic License Application (BLA) for review by FDA as they are ready, rather than waiting until the entire application is complete for FDA review. This may lead to earlier drug approval and access by patients. A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met.