The U.S. Food and Drug Administration has approved Ultragenyx Pharmaceutical's gene therapy, Fayuvi, as the first treatment for Sanfilippo syndrome Type A, a fatal inherited disorder affecting children. The therapy aims to restore sulfamidase enzyme function to clear toxic buildup in the brain and nervous system. Ultragenyx shares rose more than 10% on the news.
The approval offers a first-ever treatment option for a devastating childhood disease, potentially improving quality of life and outcomes for affected families. It also represents a significant milestone for Ultragenyx Pharmaceutical in the gene therapy space.
The U.S. Food and Drug Administration on Thursday approved Ultragenyx Pharmaceutical's gene therapy, Fayuvi, marking the first approved treatment for Sanfilippo syndrome Type A, a rare and fatal childhood disease. The inherited condition progressively damages the brain and nervous system due to a deficiency of the sulfamidase enzyme, leading to a toxic accumulation of heparan sulfate. The therapy, administered as a single intravenous infusion, works by delivering a functional copy of the SGSH gene to enable the body to produce sulfamidase and clear the damaging buildup. Clinical trials showed that children treated with Fayuvi maintained or improved cognitive function compared to untreated patients. Glenn O'Neill, president and co-founder of the nonprofit Cure Sanfilippo Foundation, whose daughter Eliza was treated with the therapy, expressed hope for a better future and quality of life for affected children. Ultragenyx expects Fayuvi to be available at specialized U.S. treatment centers within 30 to 60 days.