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 (NASDAQ: 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.

https://finance.yahoo.com/news/fda-lifts-clinical-hold-rocket-180015611.html