Rare Daily Staff
The U.S. Food and Drug Administration has placed a hold on Sarepta Therapeutics’ phase 1/2a Duchenne muscular dystrophy micro-dystrophin gene therapy trial due to the presence of a trace amount of DNA fragment in research-grade, third-party supplied plasmid.
The company said that preliminary in-vivo testing performed by the Nationwide Children’s 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 an action plan and expects to immediately submit it to the FDA for review. 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.
“We intend to rapidly respond to the FDA’s clinical hold letter, including a commitment to the agency to only use GMP-s plasmid,” said Doug Ingram, Sarepta’s president and CEO. “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.”
July 27, 2018
Photo: Doug Ingram, president and CEO of Sarepta