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NewsThe FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), the first disease-modifying gene therapy for pediatric patients with Sanfilippo syndrome type A (MPS IIIA). The one-time IV infusion delivers a working copy of the SGSH gene, enabling the body to produce the missing enzyme sulfamidase. In clinical trials, treated children showed a statistically significant 23.5-point improvement in cognitive scores compared to untreated peers.
The FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), marking a historic milestone as the first disease-modifying therapy for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A — the most common and severe form of the disease. Children with MPS IIIA lack the enzyme sulfamidase, causing heparan sulfate to accumulate in the brain and body, leading to progressive loss of cognitive, language, and developmental abilities. Symptoms typically appear between ages 1–4, and life expectancy ranges from just 11 to 19 years.
Fayuvi works by using an adeno-associated virus vector to deliver a functional copy of the SGSH gene to patients' cells, enabling them to produce sulfamidase and break down heparan sulfate. It is administered as a single intravenous infusion, with corticosteroid treatment required before and for at least 8 weeks after infusion.
By the Numbers:
Why it matters: Prior to Fayuvi, treatment only managed symptoms without altering disease course. This approval offers, for the first time, a chance to preserve cognitive function in affected children — a profound shift for families facing a devastating diagnosis.