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

The Food and Drug Administration on Thursday approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s. The therapy, called Fayuvi, was developed by Ultragenyx. It is the first drug specifically approved to treat Sanfilippo. Ultragenyx did not immediately release a price. “It’s hard to overstate what this approval would mean for everybody who is living with this really horrific disease and watching their children suffer and pass away early, and all the rest that it brings,” Cara O’Neill, chief science officer of the Cure Sanfilippo Foundation, said in an interview earlier this week. “This, gosh, would just finally mean when patients and families receive this shocking diagnosis, they wouldn’t be told to … take their kids home and love them. They would be given hope and an action plan for treatment.” STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe