Health
FDA halts Regenxbio gene therapy trial over spinal masses
The clinical trial pause follows the discovery of small spinal masses in five patients receiving a treatment for Hunter syndrome.
The short version
- The FDA has paused Regenxbio's clinical trial of a gene therapy for Hunter syndrome (MPS type II).
- Small masses were detected on the spines of five trial participants, though Regenxbio reported none of the patients showed symptoms from the masses.
- This pause comes seven months after a similar Regenxbio gene therapy for MPS type I was linked to a brain tumor in a patient.
Key facts
- The FDA initiated a clinical trial pause for Regenxbio's gene therapy targeting mucopolysaccharidosis (MPS) type II, also known as Hunter syndrome.[STAT]
- Small masses were identified on the spines of five trial participants.[STAT]
- According to Regenxbio, none of the affected patients displayed symptoms associated with the discovered spinal masses.[STAT]
- Seven months prior to this pause, a different Regenxbio therapy for MPS type I was linked to a patient developing a brain tumor, representing the first tumor conclusively connected to AAV gene therapy.[STAT]
What remains uncertain
- It remains to be seen what clinical impact or long-term safety risks the spinal masses pose to the patients, or how long the clinical trial pause will remain in place.[STAT]