Rocket Pharma announces FDA clinical hold on Phase 2 Danon gene therapy trial after patient death

ROCKET PHARMACEUTICALS, INC.

Patient in Phase 2 trial of RP-A501 experienced SAE (capillary leak syndrome) and later died due to acute systemic infection.
FDA placed clinical hold on May 23, 2025; company had voluntarily paused dosing earlier.
Root cause analysis focusing on novel immune suppression agent added to pre-treatment regimen for complement activation.
Company unable to provide guidance on trial completion timing.
Cash $318.2M as of March 31, 2025; expects to fund operations into 2027 with reduced spend.

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