一次情報の記録
決定級試験登録の変更
Rocket Pharmaceuticals:Danon病におけるRP-A501のピボタルフェーズ2試験についてFDAと合意
FDA confirmed the pivotal efficacy population of 12 male patients at the recalibrated dose of 3.8 × 10¹³ GC/kg, with the first three patients already treated counting toward this total. The agency also endorsed the 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index, intended to support a potential accelerated approval pathway. Enrollment and dosing under the modified protocol may continue, with completion of dosing expected by mid-2027.
主要事実
出典の抜粋
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,