The Sarepta Mugging and Drug Innovation

The Trump FDA tries to kill a therapy that has helped boys with a deadly diagnosis

WSJ editorial. Excerpts:

"nearly 1,000 patients have been treated with Elevidys, a large sample size for a nascent gene therapy. Both Duchenne patients who died suffered from advanced disease and could no longer walk when treated. Sarepta more than a month ago paused shipments to patients who can no longer walk."

"the FDA has demanded the company stop shipping the Duchenne drug for all patients."

"it isn’t obvious what the company can now do to receive a clean bill of health. A senior FDA official told the press last week that the drug faced an “arduous and treacherous” path back to market and that the company suspended shipments because the agency had “a gun to its head.”"

"boys improved on secondary measures such as “time to rise from the floor, 10-meter walk/run, time to ascend four steps.”"

"such secondary endpoints had been used to support prior drug approvals as primary endpoints."