FDA Lifts Clinical Hold On Rocket Pharma's X-Linked Inherited Disorder Trial

The FDA has lifted the clinical hold on Rocket Pharmaceuticals Inc's RCKT Phase 1 trial of RP-A501 (gene therapy) for 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 patient selection and management guidelines.
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 Q3 of 2021.
Related content: Benzinga's Full FDA Calendar.
The Company plans to report updated longer-term data from the low-dose (6.7e13 vg/kg) and higher-dose (1.1e14 vg/kg) young adult cohorts in Q4.
Rocket's Danon Disease program was placed on clinical hold by the FDA in May.
Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an essential mediator of autophagy.
The disorder results in the accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure.
Price Action: RCKT shares are down 0.84% at $29.67 during the market session on the last check Monday.