Regenxbio have today issued an announcement about its investigational gene therapy RGX-121 for MPS II Hunter syndrome.
The FDA in the USA has placed the trial on clinical hold after spine MRI scans found small, asymptomatic nodules or cystic mass’ in five patients who received treatment around three to six years ago. The company has said all five patients are doing well clinically, and the findings have been considered non-serious and likely benign. No nodules or masses were found on brain MRI scans.
The company has said all five patients are doing well clinically, and the findings have been considered non-serious and likely benign.
Regenxbio and its partner NS pharma are reviewing additional imaging, longer-term follow-up data and feedback from the FDA before deciding on next steps. At this stage, Regenxbio does not expect to resubmit its application to the FDA for RGX-121.
We will continue to monitor updates closely and share further information when it becomes available.
