Rocket Pharmaceuticals provides update on registration path for RP-A501

Rocket Pharmaceuticals provided an update from the company’s end-of-Phase 1 meeting with the FDA regarding RP-A501, the company’s investigational adeno-associated virus-based gene therapy for Danon Disease. During the meeting, Rocket reviewed the positive Phase 1 dataset with the FDA and proposed a study design and endpoints for ongoing clinical development of the investigational gene therapy. Based on the ongoing FDA discussion, Rocket is to pursue a biomarker-based composite endpoint for a single arm, open-label pivotal trial with a natural history comparator Initiation of initial component of global study is anticipated in 1H’23. Results from the ongoing Phase 1 Danon Disease trial show that RP-A501 was generally well tolerated with evidence of durable treatment activity and improvement of Danon Disease for both pediatric patients with up to nine months of follow-up and four adult patients with up to 36 months of follow-up.