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

The Food and Drug Administration (FDA) will reconsider approving Regenxbio's experimental gene therapy for mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, a rare and fatal childhood brain disorder. This decision marks a reversal from the FDA's rejection of the therapy just four months prior. Regenxbio's therapy aims to address the neurological form of MPS II, for which existing enzyme-replacement therapies are ineffective as they cannot penetrate the brain. This apparent turnaround by the FDA follows a pattern of reconsiderations in recent months, particularly after leadership changes within the agency. Last week, UniQure announced it received clearance to submit an application for a Huntington's disease gene therapy, which the FDA had previously rejected. The company's former commissioner, Marty Makary, had also publicly criticized the therapy.