The Food and Drug Administration will reconsider approving an experimental gene therapy for a deadly and rare childhood brain disorder that it rejected just four months ago, the therapy’s developer, Regenxbio, said on Monday.
The sudden turnaround is the latest in a series of apparent FDA reversals in the past two months, after leaders installed by the Trump administration resigned or were fired. Just last week, UniQure announced it was cleared to submit an application for a Huntington’s disease gene therapy that the agency had previously spurned and that former commissioner Marty Makary appeared to disparage on national television.
STAT Plus: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval
In Regenxbio’s case, the company had applied for approval for a gene therapy for mucopolysaccharidosis type II, also known as MPS II or Hunter syndrome. An enzyme-replacement therapy has long been available for MPS II, but the enzyme can’t enter the brain, so children with the neurological form of the disease experience progressive cognitive decline.
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