Rocket Pharma更新了RP-A501治疗达农病的进展

2026-10-07 23:44

Rocket Pharmaceuticals (RCKT) said that it has a clear path to complete a pivotal phase 2 trial for its Danon disease asset, RP-A501.
The company said 12 male patients will receive treatment. Co-primary endpoints are myocardial LAMP2 protein expression of at least Grade 1 and a reduction in left ventricular mass index of at least 10% from baseline after one year.
If met, Rocket could seek a potential accelerated approval pathway.
Phase 1 results showed that at around 12 months, the six patients in the main phase 1 analysis had myocardial LAMP2 protein expression and at least a 10% reduction in left ventricular mass index. At follow-up of three to seven years, left ventricular mass index was below baseline in five of six patients, with reductions of at least 10% in four patients.
Danon disease is a rare genetic disorder characterized by cardiomyopathy and muscle weakness.

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