FDA Lifts Clinical Hold on Sarepta’s Micro-dystrophin Gene Therapy Program for Duchenne

Rare Daily Staff

The U.S. Food and Drug Administration lifted the clinical hold on Sarepta Therapeutics’ Duchenne muscular dystrophy micro-dystrophin gene therapy program.

Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical hold due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot.

The company said that working in collaboration with Nationwide Children’s Hospital, an action plan was developed and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.

“We have been able to address the clinical hold in record time and without delay to this profoundly important clinical program,” said Doug Ingram, Sarepta’s president and chief executive officer. “Our focus now is on meeting with the Division to take guidance and gain alignment around what we hope to be our registration trial for our micro-dystrophin program and achieving our goal of commencing that trial by year-end 2018.”

September 24, 2018
Photo: Doug Ingram