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

FDA reverses course on Regenxbio’s childhood gene therapy after rejection — Live Feedhttps://www.live-feeds.com/feed/fda-reverses-course-on-regenxbio-s-childhood-gene-therapy-after-rejectionContinuously updated, source-cited coverage. FDA Reverses Rejection of Regenxbio Hunter Syndrome Gene Therapyhttps://www.live-feeds.com/feed/fda-reverses-course-on-regenxbio-s-childhood-gene-therapy-after-rejectionhttps://www.live-feeds.com/feed/fda-reverses-course-on-regenxbio-s-childhood-gene-therapy-after-rejection#u14391Fri, 26 Jun 2026 13:31:29 +0000The FDA will reconsider Navsunli, a one-time gene therapy for Hunter syndrome also known as MPS II. The agency reversed its previous demand for an untreated control arm and now backs an accelerated approval path. Regenxbio plans to resubmit its BLA in Q3 2026.What's confirmed:The FDA reversed its rejection of Regenxbio's gene therapy for Hunter syndrome.Navsunli is a one-time gene therapy for Mucopolysaccharidosis II, also known as Hunter syndrome.The FDA dropped the requirement for an untreated control arm or placebo.Regenxbio expects to resubmit the BLA in Q3 2026.This is the third rare