First-Ever Treatment for "Childhood Alzheimer's" Approved as FDA Clears Ultragenyx Gene Therapy
Families facing Sanfilippo syndrome type A now have "hope and an action plan," advocates say.
The FDA has approved Fayuvi, a gene therapy from Ultragenyx, as the first drug specifically approved to treat Sanfilippo syndrome type A, STAT reports.
Sanfilippo syndrome is an ultra-rare inherited disease, sometimes called "childhood Alzheimer's," in which children who develop normally at first gradually lose skills such as speech and walking. Until now, families have had no approved treatment.
"It's hard to overstate what this approval would mean for everybody who is living with this really horrific disease." — Cara O'Neill, chief science officer, Cure Sanfilippo Foundation
O'Neill said the approval gives families "hope and an action plan for treatment" in place of being told to take their children home.
A strong month for rare diseases
Fayuvi was one of several first-of-their-kind approvals in September:
- Zanvastro (zilganersen) from Ionis became the first FDA-approved treatment for Alexander disease, a rare neurological disorder affecting fewer than one in a million people. In its trial, treated patients walked significantly faster at 61 weeks than untreated controls. The FDA called it "a landmark moment for this community."
- Emcitate became the first FDA-approved therapy for symptoms of MCT8 deficiency, a rare genetic disease.
- Kerendia from Bayer was approved for chronic kidney disease linked to type 1 diabetes, the first new option for those patients in more than 30 years.
Ultragenyx did not immediately announce a price for Fayuvi.