Tag

Hunter Syndrome

All articles tagged with #hunter syndrome

biotech1 month ago•2 min saved

FDA hold stalls Regenxbio's RGX-121 refiling amid spine MRI findings

The FDA has placed a clinical hold on Regenxbio’s RGX-121 due to asymptomatic spine MRI findings, derailing plans to refile for approval in MPS II (Hunter syndrome). The hold follows a prior safety signal in RGX-111 and expanded monitoring that identified five patients with likely benign spinal nodules; researchers will continue imaging and use FDA feedback to determine next steps, while Regenxbio forges ahead with RGX-202 and surabgene lomparvovec. Regenxbio’s stock declined in premarket trading.

via Fierce Biotech|

#biotech #clinical-hold #fda

health1 month ago•2 min saved

FDA pauses Regenxbio Hunter syndrome gene therapy after spine masses detected in five patients

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.

via STAT|

#fda #gene-therapy #health

health3 months ago•2 min saved

FDA reopens review of Regenxbio's Hunter syndrome gene therapy

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.

via statnews.com|

#fda #gene-therapy #health

health6 months ago•2 min saved

FDA Clears Denali Drug for Hunter Syndrome Amid Tightened Rare-Disease Scrutiny

The FDA approved Denali Therapeutics' Avlayah for Hunter syndrome, marking a rare-disease drug approval at a time regulators are signaling tougher evidentiary standards after recent rejections like Regenxbio's Hunter syndrome gene therapy.

via statnews.com|

#avlayah #denali-therapeutics #fda-approval

biotech8 months ago•2 min saved

FDA rejects Regenxbio’s Hunter syndrome gene therapy over biomarker doubts

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.

via statnews.com|

#biomarker #biotech #fda

healthcare8 months ago•7 min saved

FDA nixes Regenxbio Hunter syndrome gene therapy over trial design concerns

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.

via Fierce Biotech|

#fda #gene-therapy #healthcare