Rocket Pharmaceuticals (RCKT) Gets FDA Alignment on Heart Drug Trial. Can it Ease Safety Concerns?
Rocket Pharmaceuticals (RCKT) announced FDA agreement to include 3 patients in its Phase 2 trial for Danon disease gene therapy RP-A501, with 9 more to be enrolled. The modified protocol includes dose adjustments and enhanced safety monitoring. The FDA confirmed 12-month co-primary endpoints for efficacy. The trial aims to complete dosing by mid-2027, with primary assessment 12 months post-treatment. Safety and efficacy data remain key uncertainties.






