FDA Will Reconsider Gene Therapy for Hunter Syndrome After Earlier Rejection

The Food and Drug Administration will reconsider approving an experimental gene therapy for mucopolysaccharidosis type II, also known as Hunter syndrome, the therapy’s developer Regenxbio said on Monday. Stat reported that the agency rejected the same application four months earlier. The treatment targets the neurological form of the disease.

An existing enzyme-replacement therapy cannot enter the brain, leaving children with progressive cognitive decline. The decision follows the resignation or firing of leaders installed by the Trump administration, Stat reported. Last week UniQure announced it was cleared to submit an application for a Huntington’s disease gene therapy the FDA had previously rejected.

Former FDA commissioner Marty Makary appeared to disparage that therapy on national television.