FDA Approves Avlayah for Neurologic Manifestations of Hunter Syndrome

The U.S. Food and Drug Administration approved Avlayah (tividenofusp alfa-eknm) to treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II).

“Today is a milestone day for children and their families battling Hunter syndrome,” said FDA Commissioner Marty Makary, M.D., M.P.H. “The FDA is capable of doing two things: one, exercising regulatory flexibility; and two, complying with our obligation under the law to approve drugs based on ‘substantial evidence’ of effectiveness.

We will continue to do everything we can to accelerate treatments for rare diseases.” Hunter syndrome is a rare inherited lysosomal disorder in which sugar molecules called glycosaminoglycans build up within the cells’ lysosomes. This substrate accumulation affects physical and mental development by causing abnormalities in the skeleton, heart, respiratory system, brain, and other organs.

Avlayah, an IV infusion given once weekly, is approved to treat neurologic manifestations of Hunter syndrome when the medication is started in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment.