All articles tagged with #regenxbio
The FDA has paused Regenxbio's gene therapy trial for Hunter syndrome (MPS II) after five trial participants were found to have small, asymptomatic masses on their spines, highlighting ongoing safety concerns with AAV-based gene therapies and echoing a prior brain-tumor case linked to a similar approach.
The FDA is reconsidering Regenxbio’s experimental gene therapy for mucopolysaccharidosis type II (Hunter syndrome) after previously rejecting it, part of a series of recent agency reversals amid leadership changes; the case follows another reversal where UniQure moved to submit a Huntington’s disease gene therapy previously spurned by the agency.
The FDA has rejected Regenxbio’s RGX-121 gene therapy for mucopolysaccharidosis type II (Hunter syndrome), citing concerns about the surrogate cerebrospinal fluid biomarker used to predict longer-term cognitive benefit and casting doubt on the accelerated-approval pathway.
The FDA rejected Regenxbio’s RGX-121 for Hunter syndrome, citing unresolved issues in the trial design (defining the patient population), use of a natural history control, and a biomarker surrogate endpoint, with a PDUFA date already pushed from 2025. Regenxbio plans to resubmit with longer-term data. The decision follows a broader context of safety concerns linked to Regenxbio’s RGX-111 and its brain-tumor event, but the FDA noted those issues were not cited as reasons for RGX-121’s rejection. Industry experts emphasize ongoing questions around biomarker-based approvals and vector-related safety, underscoring the urgent need for effective therapies in these ultra-rare MPS diseases.