原始出處紀錄
決策級臨床試驗登錄異動
Rocket Pharmaceuticals：FDA 就 RP-A501 用於 Danon 病的關鍵性 Phase 2 試驗達成共識
FDA 確認關鍵性療效族群為 12 名男性患者，劑量重新校準為 3.8 × 10¹³ GC/kg，前三名已接受治療的患者計入此總數。該機構亦核准 12 個月共同主要終點，包括心肌 LAMP2 蛋白表現及左心室質量指數降低 10%，旨在支持潛在加速核准途徑。修改後方案的收案與給藥可繼續進行，預計 2027 年年中完成給藥。
關鍵事實
來源摘錄
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,