First treatment approved for Sanfilippo syndrome, a fatal childhood disease
The FDA on Sept. 17 approved Fayuvi, from Ultragenyx, as the first treatment for Sanfilippo syndrome type A, a rare, fatal inherited disease that erodes children's learning, speech and movement. The one-time infusion uses a modified virus to deliver a working copy of the gene the children lack. It is approved for children whose development is still largely preserved. In the main study, 17 treated children were compared with 27 untreated children from a natural-history group; treated children scored higher on developmental tests. The company expects to ship to treatment centers within 30 to 60 days. No price was announced.
The wall: label unread; price; durability
Tier: Confirmed (approval confirmed; study figures reported (WebMD))
Sources
- fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type
- webmd.com/drugs/updates/fayuvi-sanfilippo-syndrome-type-a
- ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-approval-fayuvitm-gene-therapy-first-ever
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