Rocket Pharmaceuticals Says FDA Confirms Path to Complete Phase 2 Danon Disease Trial

07:39 AM EDT, 09/15/2026 (MT Newswires) -- Rocket Pharmaceuticals (RCKT) said Tuesday that it is aligned with the US Food and Drug Administration, or FDA, as it completes its phase 2 trial evaluating RP-A501 to treat Danon disease. The company said that the FDA reviewed data from the first three patients treated under a modified protocol and confirmed continued enrollment of 12 patients dosed at the recalibrated dose. The pivotal study will include 12 male patients, with the first three treated under the modified protocol counting toward the total. Rocket expects to enroll and dose the remaining nine patients by mid-2027. Rocket said it will conduct a primary assessment at 12 months using previously established co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index. Shares of the company were up 9% in Tuesday's premarket activity.