Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease
Rocket Pharmaceuticals announced alignment with the FDA on completing the pivotal Phase 2 trial of RP-A501 in Danon disease, with the agency confirming a 12-patient pivotal efficacy population and continued enrollment at the recalibrated dose. Initial safety data from the first three patients showed no clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome, and all three count toward the pivotal population. Nine additional patients remain to be dosed, with completion expected by mid-2027.
Key figures
- Drug Name
- RP-A501
- Phase Of Trial
- pivotal Phase 2
- Dose gc per kg
- 3.8 × 10¹³
- Program webinar
- October 6, 2026, 4:30 p.m. ET
- Patients remaining
- 9
- Pivotal population
- 12
- Co primary endpoints
- ["myocardial LAMP2 protein expression","10% reduction from baseline in left ventricular mass index"]
- Patients already dosed
- 3
- Dosing completion target
- mid-2027
- Primary assessment timepoint
- 12 months
- Accelerated approval targeted
- Yes
AI analysis
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