Home / Health / FDA Halts Regenxbio Gene Therapy Trial Over Spinal Concerns
FDA Halts Regenxbio Gene Therapy Trial Over Spinal Concerns
24 Aug
Summary
- FDA placed Regenxbio's experimental gene therapy on clinical hold.
- Spinal scans revealed abnormalities in five study participants.
- Regenxbio does not expect to resubmit the therapy's approval soon.
Regenxbio's experimental gene therapy, RGX-121, intended for treating Hunter syndrome, has been placed on clinical hold by the U.S. Food and Drug Administration. This significant decision was prompted by findings from spinal scans of five study participants who had received the therapy approximately three to six years prior.
These scans identified the presence of a small lump or fluid-filled mass in the affected individuals. Hunter syndrome, also known as MPS II, is a rare inherited disorder that can lead to progressive damage throughout the body, including the brain. In light of these developments, Regenxbio has stated that it does not expect to resubmit the application for the treatment's approval in the near term.
