FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A
Source Summary
The FDA approved rebisufligene etisparvovec (Fayuvi) as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), commonly known as Sanfilippo syndrome type A. This marks the first therapeutic option for this rare genetic disorder affecting children.
Why it matters
This approval provides the first disease-modifying treatment option for pediatric patients with a severe, progressive rare genetic disorder previously without approved therapies.
