|Other names||Vestronidase alfa-vjbk|
|Main uses||Mucopolysaccharidosis type VII (Sly syndrome)|
|Side effects||Diarrhea, rash, anaphylaxis|
|Typical dose||4 mg/kg|
|Chemical and physical data|
|Molar mass||72562.49 g·mol−1|
Common side effects include swelling at the site of injection, diarrhea, rash, and anaphylaxis. It is a recombinant form of the human enzyme beta-glucuronidase; and works by replacing this missing enzyme.
Vestronidase alfa was approved in the United States in 2017 and Europe in 2018. In the United States it costs about 2,400 USD for 10 mg as of 2021 or about 624,000 USD a year for someone who weights 25 kg.
The typical dose is 4 mg/kg every two weeks.
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. Participants received treatment with vestronidase alfa at doses up to 4 mg/kg once every two weeks for up to 164 weeks. Efficacy was primarily assessed via the six-minute walk test in ten participants who could perform the test. After 24 weeks of treatment, the mean difference in distance walked relative to placebo was 18 meters. Additional follow-up for up to 120 weeks suggested continued improvement in three participants and stabilization in the others. Two participants in the vestronidase alfa development program experienced marked improvement in pulmonary function. Overall, the results observed would not have been anticipated in the absence of treatment. The effect of vestronidase alfa on the central nervous system manifestations of MPS VII has not been determined.
The FDA approved vestronidase alfa-vjbk based primarily on evidence from one clinical trial (NCT02230566) of 12 participants with mucopolysaccharidosis VII. The trial was conducted at four sites in the United States. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.
The benefit and side effects of vestronidase alfa were based primarily on one trial. Participants were randomly assigned to four groups. Three groups of participants received placebo treatment before starting vestronidase alfa treatment and one group received vestronidase alfa only. vestronidase alfa or placebo were given once every two weeks as intravenous (IV) infusions. Neither participants nor healthcare providers knew which treatment was given until after the trial was competed.
The benefit of 24 weeks of vestronidase alfa treatment was primarily evaluated by the 6-minute walking test (6MWT) and compared to placebo treatment in ten participants who could perform the test. The 6MWT measured the distance a patient could walk on a flat surface in 6 minutes. An additional follow-up using 6MWT was done for up to 120 weeks.
The application for vestronidase alfa was granted fast track designation, orphan drug designation, and a rare pediatric disease priority review voucher. This was the twelfth rare pediatric disease priority review voucher issued.
The U.S. Food and Drug Administration (FDA) granted approval of Mepsevii to Ultragenyx Pharmaceutical, Inc, and required the manufacturer to conduct a post-marketing study to evaluate the long-term safety of the product.
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- Clinical trial number NCT02230566 for "A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)" at ClinicalTrials.gov