Vestronidase alfa, sold under brand name Mepsevii, is a medication for the treatment of Sly syndrome.
[2][8] The safety and efficacy of vestronidase alfa were established in a clinical trial and expanded access protocols enrolling a total of 23 participants ranging from five months to 25 years of age.
[5] Two participants in the vestronidase alfa development program experienced marked improvement in pulmonary function.
[5] The effect of vestronidase alfa on the central nervous system manifestations of MPS VII has not been determined.
[5] The FDA approved vestronidase alfa-vjbk based primarily on evidence from one clinical trial (NCT02230566) of 12 participants with mucopolysaccharidosis VII.
[5] The US Food and Drug Administration (FDA) granted approval of Mepsevii to Ultragenyx Pharmaceutical, Inc,[5] and required the manufacturer to conduct a post-marketing study to evaluate the long-term safety of the product.