FDA lifts clinical hold on Rocket gene therapy trial, creating launchpad for start of pediatric dosing

Rocket Pharmaceuticals’ gene therapy trial is back on track. With the FDA lifting the clinical hold after Rocket changed the study protocol, the biotech is now on course to start dosing children in the third quarter.

Late last year, Rocket reported data from a phase 1 trial of its gene therapy treatment for Danon disease, a genetic disorder that can lead to heart failure and death in adolescence or early adulthood. The results sparked a surge in Rocket’s share price but also revealed a drug-related serious adverse event in the higher-dose cohort.

Months later, the FDA slapped a clinical hold on Rocket’s Danon program. Rocket framed the action as a brief pause needed to revise the guidelines for patient selection and management and targeted the resumption of treatment in the third quarter.

Rocket looks set to deliver on that target. The FDA recently lifted the hold after Rocket addressed its requests to modify the trial protocol and other supporting documents, sparking work to resume the program. Rocket aims to start dosing in the low-dose pediatric cohort in the third quarter.

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“Given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the pediatric cohort,” Rocket CEO Gaurav Shah said in a statement. “This is another important step forward as we believe the pediatric Danon population has the potential to realize the maximum benefit from our Danon Disease gene therapy program.”

As Rocket revealed last week, the high dose is no longer part of its plans. The removal of the high dose from the plans follows the serious adverse event seen in that cohort and the generation of data on the magnitude and durability of the effect seen at the low dose. Focusing on the low dose could enable Rocket to reduce the size of its phase 1 trial and move more quickly into phase 2.


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