Skip to main content
Premium Trial:

Request an Annual Quote

Duchenne Trial Halted

Federal regulators have halted Sarepta Therapeutics' trial of its gene therapy to treat Duchenne muscular dystrophy, Stat News reports. The treatment, it adds, provides a smaller version of the dystrophin gene to patients who lack it.

The company released preliminary results last month from three children in its trial that looked promising, CNBC reported at the time. The company said patients who received its drug exhibited an 87 percent decrease in their creatine kinase levels after two months, according to CNBC.

But US Food and Drug Administration officials have now stopped the trial, which is being conducted at Nationwide Children's Research Institute, after a DNA fragment was found in a plasmid where it should not have been, MarketWatch adds. Sarepta says that the misplaced fragment was found during routine quality assurance testing, and Nationwide says that lot had not been used in patients, according to Stat News.

"Patient safety is our top priority at Sarepta as we know it is for Nationwide Children's Research Institute," says Sarepta President and CEO Doug Ingram in a statement.

MarketWatch adds that, if FDA accepts its proposal to address the problem, Sarepta plans to begin dosing patients by the end of the year.