FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease — Live Feedhttps://www.live-feeds.com/feed/fda-approves-a-new-gene-therapy-for-sanfilippo-syndrome-an-ultra-rare-diseaseContinuously updated, source-cited coverage. FDA Approves Fayuvi Gene Therapy for Sanfilippo Syndromehttps://www.live-feeds.com/feed/fda-approves-a-new-gene-therapy-for-sanfilippo-syndrome-an-ultra-rare-diseasehttps://www.live-feeds.com/feed/fda-approves-a-new-gene-therapy-for-sanfilippo-syndrome-an-ultra-rare-disease#u77331Sun, 20 Sep 2026 10:35:05 +0000The Food and Drug Administration approved Ultragenyx Pharmaceutical's gene therapy Fayuvi to treat Sanfilippo syndrome Type A, marking the first-ever treatment for the rare neurodegenerative condition. Following the approval, the company set the price for the gene therapy at $3.95 million. Investor reaction drove Ultragenyx stock up 13 percent. The approval also includes a Priority Review Voucher for the company. This marks a major milestone for patients with the fatal pediatric disorder who previously had no approved treatments available on the market.Why it mattersSanfilippo syndrome is