Posted by Michael Wonder on 07 Feb 2023
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-A501 gene therapy for Danon disease
7 February 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to RP-A501, the Company’s investigational adeno-associated virus based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available.
Regenerative medicine advanced therapy 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 through the program’s development.
