RCKT

Announcements

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Daily updates

• Rocket Pharmaceuticals established an FDA-agreed pivotal Phase 2 strategy for RP-A501 in Danon disease, with 12 male patients to receive an adjusted dosing regimen.Oct 8, 2026
• Rocket Pharmaceuticals reached FDA alignment on the Phase 2 RP-A501 trial design in Danon diseaseSep 15, 2026

FDA designations

• Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A601 Gene Therapy for PKP2-Arrhythmogenic CardiomyopathyJul 21, 2025

News

• Rocket Pharmaceuticals announced FDA alignment on the path to completing the pivotal Phase 2 trial of RP-A501 in Danon diseaseSep 15, 2026
• Rocket Pharmaceuticals reported a positive safety update from the first three patients treated with RP-A501 under the modified Phase 2 protocolAug 3, 2026
• Rocket Pharmaceuticals received FDA approval for KRESLADI to treat pediatric patients with LAD-IMar 27, 2026
• Rocket Pharmaceuticals announces FDA acceptance of BLA resubmission of KRESLADI™ for treatment of severe leukocyte adhesion deficiency-I (LAD-I)Oct 14, 2025
• Rocket Pharmaceuticals announces FDA has lifted clinical hold on pivotal Phase 2 trial of RP-A501 for treatment of Danon diseaseAug 20, 2025