Rocket Pharma says first three patients dosed with RP-A501 in modified Phase 2 Danon trial show favorable safety profile
RCKT•FDA engagement and next steps
The company is engaging with the FDA on a path to resume broader dosing and complete the 12-patient, single-arm pivotal study.
A regulatory pathway update is expected in the second half of 2026, with a broader program update also slated for the second half of 2026.
Early safety update from Danon trial
Rocket Pharmaceuticals reported a positive early safety update from the first three patients treated in its pivotal Phase 2 RP-A501 trial for Danon disease.
Results have already been observed, showing treatment was well tolerated with no major safety signals reported in these initial patients.
