FDA Reverses on Regenxbio Hunter Syndrome Gene Therapy
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- Regenxbio said the FDA will reconsider approving its gene therapy for mucopolysaccharidosis type II (Hunter syndrome), a rare and deadly childhood brain disorder the agency rejected four months ago.
- The existing enzyme-replacement therapy for MPS II cannot cross into the brain, leaving children with the neurological form facing progressive cognitive decline.
- The FDA's reversal is the latest in a string of agency about-faces in the past two months, following the resignations or firings of FDA leaders installed by the Trump administration.
- Last week, UniQure announced it was cleared to resubmit a Huntington's disease gene therapy the FDA had previously spurned — a decision former commissioner Marty Makary had appeared to disparage on national television.
Why it matters: Children with the neurological form of MPS II currently have no treatment that reaches the brain, making the FDA's reconsideration of Regenxbio's gene therapy a potential lifeline against progressive cognitive decline. Two rejections overturned in as many weeks — including UniQure's Huntington's therapy — suggests a systematic shift in how the agency weighs rare-disease applications after its leadership turnover.
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