On Tuesday, Rocket Pharmaceuticals Inc (NASDAQ: RCKT) announced that alignment has been reached with the FDA on the global Phase 2 pivotal trial of RP-A501 for Danon Disease.
Danon Disease is an inherited cardiomyopathy that leads to mortality in most male patients at age ~20 and females at age ~40.
The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.
To support ...