(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 said.
The patient in the May trial had suffered from capillary leak syndrome that causes swelling and low blood pressure.
Danon disease is a rare genetic disorder, leading to heart muscle damage and progressive muscle weakness.
(Reporting by Kamal Choudhury and Padmanabhan Ananthan in Bengaluru; Editing by Sahal Muhammed)
Comments