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Rocket Pharmaceuticals: nagkasundo sa FDA hinggil sa pivotal Phase 2 trial ng RP-A501 para sa Danon disease

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.

Mahahalagang fact

Na-file
Set 15, 2026, 11:06 AM
Kaganapan
Pagbabago sa trial registry
Direksyon
Positibo
Kumpanya
RCKTRocket Pharmaceuticals
Asset
RP-A501
Pinagmulan
SEC 8-K
Kumpiyansa
May source
Dosis
3.8 × 10¹³ GC/kg
Pangunahing populasyon
12 pasyente
Magkasamang pangunahing layunin
Pagpapahayag ng LAMP2 protein sa kalamnan ng puso at 10% na pagbawas sa left ventricular mass index sa 12 buwan

Sipi mula sa source

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,

SEC 8-K

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