FDA Lifts Rocket Pharmaceuticals Study Hold For Rare Disease Gene Therapy

Core Insights - Rocket Pharmaceuticals Inc. experienced a significant increase in stock price following the FDA's decision to lift the clinical hold on its Phase 2 trial for RP-A501 targeting Danon disease [1][5] - The clinical hold was resolved in under three months, allowing the trial to resume with a recalibrated dosing strategy [1][3] Company Developments - The FDA's hold was initially placed due to a patient death linked to an acute systemic infection during the study [2] - Rocket Pharmaceuticals has addressed the FDA's concerns, leading to the resumption of the pivotal study with a new dose of 3.8 x 10¹³ GC/kg of RP-A501 [3][4] - The adjusted dosing aligns with the efficacy observed in the Phase 1 study and aims to ensure safety and efficacy in the ongoing trial [4] Treatment Protocol Adjustments - The revised treatment regimen will discontinue the prophylactic use of a C3 complement inhibitor while retaining sirolimus, rituximab, and steroids [5] - A lower threshold for administering a C5 inhibitor (eculizumab) will be specified to manage complement activation [5] - To date, six patients with Danon disease have been treated in the Phase 2 study with RP-A501 [5]