原始来源记录
决策级临床试验登记变更
Rocket Pharmaceuticals：FDA就Danon病RP-A501关键II期试验达成一致
FDA确认关键疗效人群为12名男性患者，重新校准剂量为3.8 × 10¹³ GC/kg，前三名已接受治疗的患者计入该总数。FDA还认可12个月共同主要终点，即心肌LAMP2蛋白表达和左心室质量指数降低10%，旨在支持潜在的加速批准途径。修改方案下的入组和给药可继续进行，预计2027年年中完成给药。
关键事实
来源摘录
EX-99.1 2 ef20082010_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease FDA confirms pivotal efficacy population of 12 patients, including the first three treated under the modified protocol FDA review of initial safety data supports continued enrollment and dosing under modified protocol FDA confirms established 12-month co-primary endpoints intended to support potential accelerated approval Comprehensive Danon disease program webinar for October 6, 2026, at 4:30 p.m. ET CRANBURY, N.J. – September 15, 2026 – Rocket Pharmaceuticals, Inc . (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and life-threatening diseases,