Marnetegragene Autotemcel Application Receives Extended Review Period

The US Food and Drug Administration (FDA) has extended the Priority Review period for the Biologics License Application (BLA) by three months for marnetegragene autotemcel (KRESLADI™) as a treatment for patients with severe leukocyte adhesion deficiency-I (LAD-I), according to a press release by Rocket Pharmaceuticals, the manufacturer of the drug.

The BLA for marnetegragene autotemcel was supported by data from a phase I/II study that reported a 100% overall survival rate in the 12 months after infusion among nine patients with LAD-I. Researchers also observed “large decreases” in the incidence of significant infections and improvements in skin lesions and wound repair capabilities. The study met all primary and secondary endpoints and described marnetegragene autotemcel as very well tolerated with no treatment-related serious adverse events.

“We look forward to continuing our close collaboration with the FDA and together share a deep sense of responsibility in the rigorous process required to bring novel, potentially curative gene therapies, like KRESLADI™ to patients who need them most,” said Gaurav Shah, MD, Chief Executive Officer at Rocket Pharmaceuticals. “We remain confident and focused on making this therapy available for patients as quickly as possible.”

The review period was extended to June 30, 2024, to review additional information requested by the FDA on Chemistry, Manufacturing, and Controls. There will not be an advisory committee meeting.