The FDA has placed a clinical hold on Regenxbio’s gene therapy, RGX-121, for mucopolysaccharidosis II, also known as Hunter syndrome, after spinal MRIs turned up asymptomatic nodules or cystic masses in five participants in the Campsiite trial.
The findings were identified through an expanded MRI monitoring plan Regenxbio put in place following an earlier hold on its related MPS I therapy, RGX-111. Investigators consider the findings nonserious, and radiologists believe they are likely benign, though Regenxbio said there is no clinical or pathological evidence yet confirming their cause. No abnormalities were found on brain MRIs, according to an Aug. 24 news release.
Regenxbio does not expect to resubmit its biologics license application for RGX-121 “in the near term.” The FDA had issued a complete response letter for the therapy in February, citing gaps in the evidence supporting effectiveness.
At the Becker’s 32nd Annual Meeting: The Business and Operations of ASCs, taking place October 29-31 in Chicago, ASC leaders, surgeons and healthcare executives will explore strategies to drive growth, enhance operational performance, navigate reimbursement challenges and prepare for the future of ambulatory surgery. Apply for complimentary registration now.
