FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf) on September 17, 2026, as the first treatment for mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) in pediatric patients. Fayuvi is a one-time intravenous gene therapy using adeno-associated virus serotype 9 to deliver a functional SGSH gene. The approval provides a treatment option for a rare pediatric genetic disease previously without FDA-approved therapies. State Medicaid programs and managed care plans will need to determine coverage and reimbursement policies for this specialty gene therapy.
State Medicaid agencies and MCOs must establish prior authorization criteria, medical necessity guidelines, and negotiate pricing for a high-cost specialty gene therapy targeting a rare pediatric population eligible for Medicaid coverage under EPSDT.
Pharmacy · Managed Care
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