Vestronidase alfa, sold under brand name Mepsevii, is a drug for the treatment of Sly syndrome.[2] 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.[3][4] MPS VII is an extremely rare, progressive condition that affects most tissues and organs.[3]
The most common side effects after treatment with vestronidase alfa include infusion site reactions, diarrhea, rash (urticaria) and anaphylaxis (sudden, severe allergic reaction).[3][5]
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[6] It was approved for use in the European Union in August 2018.[5]
^"FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
^McCafferty EH, Scott LJ (April 2019). "Vestronidase Alfa: A Review in Mucopolysaccharidosis VII". BioDrugs. 33 (2): 233–240. doi:10.1007/s40259-019-00344-7. PMC 6469592. PMID 30848434.
^ abc"FDA approves treatment for rare genetic enzyme disorder" (Press release). U.S. Food and Drug Administration (FDA). 15 November 2017. Archived from the original on 10 December 2019. Retrieved 9 December 2019. This article incorporates text from this source, which is in the public domain.
^"Drug Trial Snapshot: Mepsevii". U.S. Food and Drug Administration (FDA). 4 December 2017. Archived from the original on 10 December 2019. Retrieved 9 December 2019. This article incorporates text from this source, which is in the public domain.
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