The pharmacokinetics and pharmacodynamic characteristics of vestronidase alfa, the first and only specific treatment for mucopolysaccharidosis VII (Sly Syndrome; an ultra-rare inherited lysosomal disorder) approved in the US and EU were reported for the first time.

Results of modeling and simulations supported the recommended body weight-based dosing regimen of vestronidase alfa, which is 4 mg/kg every other week by infusion regardless of age.

A higher dose likely will not further increase the treatment benefit, whereas reducing dosing frequency to once every 4 weeks may result in partial loss of efficacy, even if the total monthly dose amount is maintained at 8 mg/kg.