Rocket Gets RMAT Designation From FDA For RP-A501 Therapy Targeting Danon Disease

(RTTNews) - Late-stage biotechnology company Rocket Pharmaceuticals, Inc. (RCKT) announced Tuesday that FDA has granted Regenerative Medicine Advanced Therapy or RMAT designation to RP-A501, adeno-associated virus-based gene therapy for the treatment of Danon Disease.

The company noted that RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A501 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review.

Rocket Pharma is planning to initiate the Phase 2 trial of RP-A501 in the second quarter of 2023.

Danon Disease is a rare X-linked inherited genetic cardiac disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2. There are no disease-altering therapies available.