by: Parent Project Muscular Dystrophy
We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne 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.
In response, and 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.
PPMD, with the help of this community, was proud to support the early micro-dystrophin gene therapy work of Drs. Mendell and Rodino-Klapac. We continue to hope that gene therapy will be a potential therapy to end Duchenne and want to again thank the families participating in this cutting edge trial.
Read the press release from Sarepta:
Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
CAMBRIDGE, Mass., Sept. 24, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that the Food and Drug Administration (FDA) has lifted the clinical hold for the Company’s Duchenne muscular dystrophy (DMD) 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. In response, and 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.
“Thanks to the diligent and rapid work of my Sarepta colleagues and Nationwide Children’s Hospital in compiling and submitting a complete response and the expeditious evaluation by the FDA in reviewing the response and removing this clinical hold, we have been able to address the clinical hold in record time and without delay to this profoundly important clinical program,” stated 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.”
