REGENXBIO pauses RGX-121 FDA review

• The FDA placed a clinical hold on REGENXBIO’s RGX-121 gene therapy program after spine MRI findings in five CAMPSIITE study participants.
• REGENXBIO stated that affected participants remain clinically well, with stable or improved neurocognitive and neurobehavioral assessments.
• The company does not expect to resubmit the RGX-121 BLA in the near term and will evaluate additional data with regulators.

REGENXBIO (NASDAQ:RGNX) reported that the U.S. Food and Drug Administration placed a clinical hold on its investigational MPS II gene therapy RGX-121 following spine MRI findings in five participants in the CAMPSIITE study.

The findings involved small spinal nodules or cystic masses identified in participants who received RGX-121 through intracisternal or intraventricular dosing approximately three to six years ago.

REGENXBIO stated that investigators considered the findings nonserious and radiologists believe the abnormalities are likely benign, while no brain nodules or masses were observed.

The company stated that it does not expect to resubmit the RGX-121 Biologics License Application in the near term.

REGENXBIO and partner NS Pharma are reviewing additional imaging and long-term follow-up data while incorporating FDA feedback into future plans.

The company continues advancing other programs, including a planned Duchenne gene therapy BLA submission in the current quarter and wet age-related macular degeneration pivotal trial topline data expected in the fourth quarter.