FDA halts Rocket Pharma's gene therapy trial as complications lead to patient death
May 27 (Reuters) - The U.S. FDA has paused a mid-stage gene therapy trial by Rocket Pharmaceuticals (RCKT.O), opens new tab after a patient suffered serious complications, which ultimately led to the person's death, the drug developer said on Tuesday.
Shares of the New Jersey-based company tumbled 63% to $2.30 in premarket trading. They had closed at $6.27 on Friday.
The company said the FDA had placed a hold on the trial on Friday after the report of a capillary leak syndrome, a condition where fluid and proteins leak out of tiny blood vessels into surrounding tissues, causing swelling and low blood pressure. The patient had subsequently died, it said.
Rocket was testing its experimental gene therapy, RP-A501, for the treatment of Danon disease, a rare genetic disorder that causes heart muscle damage and progressive muscle weakness.
The company said it had voluntarily paused further dosing in the study. It is also working with the U.S. Food and Drug Administration and experts to resolve the trial halt and resume the study quickly, but cannot provide a timeline for completion until the clinical hold is lifted.
The FDA did not immediately respond to a Reuters request for comment.
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