FDA Lifts Hold on Rocket Pharma’s Danon Disease Therapy

The FDA has lifted its clinical hold on Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, an ultra-rare genetic disorder that can lead to heart failure in children and young adults.

The therapy, called RP-A501, had its phase 1 trial placed on hold by the FDA in May of this year, when the agency asked the company to modify the study protocol and supporting documents with revised guidelines for patient selection and safety management.

The three-month 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.

Rocket said it has now initiated steps to resume the program and expects to start dosing in the low-dose pediatric patient cohort in the third quarter.