STAT+: FDA reverses course on Regenxbio’s childhood gene therapy after rejection

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.&#x A0; 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, Uni Qure 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. 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. Continue to STAT+ to read the full story…