The FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A.
Fayuvi is a one-time intravenous gene therapy that uses adeno-associated virus serotype 9 to deliver a working copy of the SGSH gene into patients’ cells, according to a Sept. 17 news release. The FDA granted the approval to Ultragenyx Pharmaceutical.
The therapy’s safety and effectiveness were evaluated in an open-label, single-arm, multicenter clinical study. Patients ages 2 to 5 who received Fayuvi maintained or improved cognitive function compared with an untreated historical control cohort.
Adverse reactions reported in more than 5% of patients included increased liver enzymes, nausea and vomiting, fever, decreased appetite, decreased white blood cell and platelet counts and increased amylase. The therapy carries a warning for the risk of thrombotic microangiopathy.
The FDA granted Fayuvi orphan drug, fast track and breakthrough therapy designations.
