Danon Disease is a fatal genetic cardiac disease for which there are no disease-altering treatments currently available.
RMAT designation was granted by the regulatory body based on positive safety and efficacy data from the Phase 1 RP-A501 clinical trial.
This designation will offer the benefits of added intensive FDA guidance and accelerated review through the programme’s development.
Rocket Pharmaceuticals CEO Gaurav Shah said: “The exciting RMAT designation demonstrates recognition from the FDA of the early meaningful benefit of RP-A501 in Danon Disease and its potential to deliver lifesaving treatment for patients.
“We look forward to the FDA’s added guidance and support on the most efficient development and approval pathway for RP-A501, including on our anticipated Phase 2 pivotal trial with the opportunity for accelerated approval.”
Dr. Shah continued, “RP-A501 is the first cardiac gene therapy to receive RMAT designation from the FDA, and today’s news is another important step forward both for patients with Danon Disease and for the gene therapy field. We remain on track to initiate our Phase 2 trial in the second quarter and are thankful to the FDA for their continued collaboration.”
Rocket also holds Orphan Drug (US) and Rare Pediatric designations for the RP-A501 programme.
The initiation of the Phase II trial is on track for Q2 2023.
Rocket expects to pursue an open-label, single arm trial with a biomarker-based composite endpoint and a natural history comparator.