Sarepta: Phase 1/2a DMD Micro-Dystrophin Gene Therapy trial placed on hold
Sarepta Therapeutics has been notified by the Research Institute at Nationwide Children's Hospital that they have received a letter from the Food and Drug Administration on July 24, 24 stating that their Phase 1/2a Duchenne Muscular Dystrophy Micro-Dystrophin Gene Therapy Trial has been placed on clinical hold due to the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. Preliminary in-vivo testing performed by the Research Institute indicates that the trace fragment does not result in protein expression and is quickly cleared.The Research Institute, working with Sarepta, has developed their action plan with immediate plans to submit for review by the FDA, which will include the use of GMP-s plasmid for the program. Subject to the FDA's acceptance of the action plan, Sarepta does not anticipate any material delay in dosing patients as originally planned by year-end 2018."Patient safety is our top priority at Sarepta as we know it is for Nationwide Children's Research Institute," stated Doug Ingram, Sarepta's president and chief executive officer. "We intend to rapidly respond to the FDA's clinical hold letter, including a commitment to the Agency to only use GMP-s plasmid. Independently, we will also request a meeting with the Agency to discuss the micro-dystrophin program with the goal of commencing a pivotal trial by year-end 2018."