FDA Approves First-Ever Gene Therapy for Sanfilippo Syndrome Type A

The FDA has approved Fayuvi, a groundbreaking gene therapy developed by Ultragenyx Pharmaceutical Inc. (RARE), for the treatment of Sanfilippo syndrome Type A. This marks the first-ever treatment targeting the root cause of this rare inherited disease that progressively destroys the brain and nervous system in children.

Fayuvi is a one-time infusion administered through a modified virus, which allows it to reach the brain and central nervous system. Clinical trial data showed that children who received the therapy scored 23.5 points higher on cognitive tests compared to untreated patients. The FDA based its approval on up to eight years of clinical trial data.

Ultragenyx's stock price surged 13% to $14.50 on Thursday following the announcement. This is the company's second gene therapy approval and sixth FDA approval overall. Fayuvi will only be available at Qualified Treatment Centers, which are specialized hospitals trained to administer gene therapies.