Home » FDA Pauses Early-Stage Trial of Rocket Pharmaceuticals’ Gene Therapy
FDA Pauses Early-Stage Trial of Rocket Pharmaceuticals’ Gene Therapy
Drugs Regulatory Affairs
The FDA has temporarily paused Rocket Pharmaecuticals’ phase 1 trial evaluating an investigational gene therapy for Danon disease, a rare life-threatening heart condition.
The agency has asked for additional supporting documents on risk mitigation and revised guidelines on patient selection and management.
The temporary pause was not prompted by drug-related adverse events in either the low- or high-dose adult arms of the phase 1 study, the company said.
“We anticipate a one-quarter delay in enrollment to address FDA requests for risk mitigation methods in our protocol,” said Gaurav Shah, CEO of Rocket Pharmaceuticals.