Rocket Pharma gets FDA agreement on pivotal Phase 2 design for RP-A501 in Danon disease

 

Rocket Pharma gets FDA agreement on pivotal Phase 2 design for RP-A501 in Danon disease, supporting potential accelerated approval

• Pivotal Phase 2 trial will enroll a 12-patient efficacy population for RP-A501 in Danon disease.
• FDA agreed to continued dosing in the study under a modified clinical protocol.
• Co-primary efficacy and safety endpoints at 12 months are intended to support potential accelerated approval.

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