Sarepta DMD gene therapy hold lifted in ‘record time’

DNA genomics precision medicine
Sarepta is hoping its gene therapy will treat more patients with the disease. (Pixabay)

Two months after Sarepta was hit with a clinical hold by the FDA, the rare disease biotech has seen this lifted after sorting out a quality blip.

Back in July, the phase 1/2a trial Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) was placed on hold by the regulator after rogue DNA was found in a test sample.

The FDA sent a letter to Nationwide Children’s Hospital, which is carrying out the trial, saying it had detected a trace fragment of DNA plasmid in one lot of the microdystrophin gene therapy material, according to Sarepta CEO Doug Ingram.


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On a conference call at the time, Ingram said the lot came from research-grade plasmid material supplied by another company for use in the gene therapy, but had not been used in patients. An earlier lot from the same supplier had been used in four patients but seems to have no safety issues as the fragment hasn’t shown up in patient biopsies.

Ingram added on the call: “We know the source of the problem, we have been given clear guidance on how to address the issue, and we have a plan to allow our clinical development program to remain on track.”

RELATED: Sarepta leaps 50% on early data for Duchenne gene therapy

And it appears this was so, as today Sarepta says the hold has been lifted. “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,” said Ingram today.

“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.” 

Back in June, shares in the company leaped 50% on the back of preliminary data from three patients in the study, which revealed an increase in microdystrophin—a truncated form of dystrophin—which it said was large enough to indicate that the therapy might stop, or even reverse, DMD progression.

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