FDA, Regulatory

FDA lifts clinical hold on Sarepta’s Duchenne MD gene therapy, clearing the way to a quick launch of a pivotal trial

In a swift turnaround, Sarepta announced that it has cleared the FDA’s concerns about its gene therapy program for Duchenne muscular dystrophy, convincing regulators to lift a clinical hold issued just two months ago.

Doug Ingram

Together with collaborators at the Nationwide Children’s Hospital, the company submitted an action plan to address the “presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot” — which they say triggered the hold. The partners offered up “an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.”

CEO Doug Ingram is clearly pleased with the progress, seeing as the FDA accepted the plan “in record time” without material delay, just as he anticipated back in July.

“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,” he said in a statement.

Shares of the Cambridge, MA-based biotech $SRPT climbed 2.25% in pre-market trading.

This is a program that took investors by pleasant surprise when Sarepta rolled out some stellar, if early, data on three boys enrolled in their Phase I study.

Mean gene expression as measured by percentage of micro-dystrophin positive fibers was 76.2% and the mean intensity of the fibers was 74.5% compared to normal control. When you break that down by patient, the percentages on micro-dystrophin expression in muscle fibers varied a bit, ranging from a low of 59% to 83%, while the percentage of dystrophin-positive fibers fell in a more narrow band of 73.5% to 78%.

Sarepta believes their regulatory journey here can take as little as two years — provided no more hiccups happen. And that would mark the first pivot into gene therapy, a field it’s been keen to get deeper into as demonstrated in a $30 million deal to acquire a group of CNS therapies from University of Florida spinout Lacerta.


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