Clinical data | |
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Trade names | Mepsevii |
Other names | Vestronidase alfa-vjbk |
AHFS/Drugs.com | Monograph |
License data | |
Routes of administration | Injection |
ATC code | |
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Legal status | |
Identifiers | |
CAS Number | |
UNII | |
KEGG | |
Chemical and physical data | |
Formula | C3308H4996N874O940S16 |
Molar mass | 72562.49 g·mol−1 |
Vestronidase alfa, sold under brand name Mepsevii, is a medication for the treatment of Sly syndrome.[4] It is a recombinant form of the human enzyme beta-glucuronidase. It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.[5][6] MPS VII is an extremely rare, progressive condition that affects most tissues and organs.[5]
The most common side effects after treatment with vestronidase alfa include infusion site reactions, diarrhea, rash (urticaria) and anaphylaxis (sudden, severe allergic reaction).[5][2]
The US. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7] It was approved for use in the European Union in August 2018.[2]