US FDA lifts clinical hold on Rocket Pharmaceuticals’ gene therapy trial

(Reuters) -Rocket Pharmaceuticals said on Wednesday that the U.S. Food and Drug Administration has lifted the clinical hold on a mid-stage trial of its gene therapy, less than three months after it was halted due to a patient’s death.

Rocket Pharma’s trial for its experimental gene therapy, RP-A501, for a genetic disorder called Danon disease was halted in May by the FDA after a patient died from serious complications.

The FDA cleared the trial to resume with a lower dose than the May trial, with three patients treated one after another, at least four weeks apart. The company said the adjusted dose matches levels that showed benefit and a better safety profile in a earlier study.

The FDA confirmed that Rocket satisfactorily addressed issues outlined in the clinical hold, the company sai