Rebisufligene etisparvovec

FAYUVI is indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. FAYUVI is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function.

FAYUVI is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to deliver a functional copy of the human N-sulfoglucosamine sulfohydrolase ( SGSH ) gene to cells. Intravenous infusion of FAYUVI results in production of the lysosomal enzyme sulfamidase in target tissues with subsequent breakdown of heparan sulfate (HS) in lysosomes.

For single-dose intravenous infusion only. Review baseline liver function tests and assess platelet counts, prothrombin time, and activated partial thromboplastin time prior to FAYUVI infusion. The recommended dose of FAYUVI is 3.0 × 10 13 vector genomes (vg) per kg body weight. Starting 1 day prior to infusion, administer corticosteroids for a minimum of 8 weeks. Administer FAYUVI as a slow intravenous infusion over approximately 1 hour.

None. None.

Hepatotoxicity: Monitor liver enzymes and total bilirubin levels until 2 weeks after the corticosteroid taper is complete and as clinically indicated. Continue to monitor patients who develop liver enzyme elevations until liver enzymes return to baseline. Thrombocytopenia: Monitor platelet counts weekly for the first 4 weeks, then monthly for 6 months following infusion. Thrombotic Microangiopathy (TMA): If signs and symptoms occur, promptly undertake further diagnostic evaluation for hemolytic anemia and renal dysfunction. Hypersensitivity and Infusion Reactions: Monitor during and after infusion. If symptoms occur, pause the infusion. If appropriate, restart at a slower rate after the infusion reaction has resolved. Risk of Malignancy: In the event that a malignancy occurs, contact Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.

Most common adverse reactions (≥5%): liver enzyme increased, vomiting, pyrexia, white cell count decreased, decreased appetite, platelet count decreased, nausea, amylase increased.

Vaccinations Prior to FAYUVI administration, consider the patient's vaccination status. Vaccines should be avoided 30 days prior to treatment with FAYUVI (and use of corticosteroids) and while on corticosteroid therapy. A patient's vaccination schedule may need to be adjusted to accommodate corticosteroid administration prior to and following FAYUVI infusion.

Store upright at ≤ -60°C [-76°F]. Once drawn from the vial and prepared in syringes, FAYUVI can be stored at room temperature (20°C – 25°C [68°F – 77°F]) for up to 24 hours, including time for infusion. If necessary, an intact vial (stopper not yet punctured) that has been thawed can be stored refrigerated (2°C – 8°C [36°F – 46°F]) for up to 12 days, upright and protected from light (e.g., in the original carton). Store in the original carton until ready to use.