Rocket Pharmaceuticals says FDA aligns on RP-A501 Danon disease pivotal trial path
Rocket Pharmaceuticals said the U.S. Food and Drug Administration confirmed continued enrollment and dosing under a modified protocol for its global pivotal Phase 2 trial of RP-A501 in Danon disease, along with a 12-patient pivotal population and established 12-month co-primary endpoints. The alignment is a regulatory milestone for the program, and the company said it expects to complete dosing of the remaining patients by mid-2027.
Key points
- FDA confirmed a pivotal population of 12 male patients treated at the recalibrated dose of 3.8 × 10¹³ GC/kg.
- The first three patients treated under the modified protocol count toward the 12-patient pivotal population, leaving nine to be enrolled and treated.
- Primary assessment at 12 months uses myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index as co-primary endpoints.
- Rocket plans a Danon disease program webinar on October 6, 2026, at 4:30 p.m. ET.
The filing
Written by AI on Nov 9, 2026, 8:36 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on September 15, 2026.
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