Rocket Pharma Says FDA Lifts Clinical Hold On Phase 1 Trial Of RP-A501 For Danon Disease

(RTTNews) - Rocket Pharmaceuticals, Inc. (RCKT) announced Monday that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume.

The hold was removed after the Company addressed the FDA's requests to modify the trial protocol and other supporting documents with revised guidelines for patient selection and management.

Rocket's Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.

The Company has initiated steps to resume the program as soon as possible and expects to commence dosing in the low-dose (6.7e13 vg/kg) pediatric patient cohort in the third quarter.

The company continues to anticipate reporting updated longer-term data from the low-dose (6.7e13 vg/kg) and higher-dose (1.1e14 vg/kg) young adult cohorts in the fourth quarter.

The non-randomized, open-label Phase 1 trial is designed to enroll both pediatric and young adult male patients to assess the safety and tolerability of a single intravenous (IV) infusion of RP-A501.