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FDA to reconsider Regenxbio Hunter syndrome gene therapy
statnews.com • • business
Key Points:
- The FDA will reconsider approval of Regenxbio's experimental gene therapy for mucopolysaccharidosis type II (MPS II), a rare and deadly childhood brain disorder, after previously rejecting it four months ago.
- This reversal follows a pattern of recent FDA decisions changing under new leadership after the departure of Trump-era officials.
- Regenxbio's gene therapy targets the neurological form of MPS II, which current enzyme-replacement treatments cannot effectively treat because they do not cross the blood-brain barrier.
- The reconsideration aligns with similar developments, such as UniQure being allowed to submit an application for a Huntington’s disease gene therapy previously rejected by the FDA.
