Vestronidase alfa

Vestronidase alfa
Clinical data
Trade namesMepsevii
Other namesVestronidase alfa-vjbk
AHFS/Drugs.comMonograph
License data
Routes of
administration		Injection
ATC code
Legal status
Legal status
Identifiers
CAS Number
UNII
KEGG
Chemical and physical data
FormulaC3308H4996N874O940S16
Molar mass72562.49 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]

  1. ^ "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.
  2. ^ a b c "Mepsevii EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 28 February 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  3. ^ "Mepsevii PI". Union Register of medicinal products. 27 August 2018. Retrieved 29 June 2024.
  4. ^ 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.
  5. ^ a b c "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. Public Domain This article incorporates text from this source, which is in the public domain.
  6. ^ "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. Public Domain This article incorporates text from this source, which is in the public domain.
  7. ^ New Drug Therapy Approvals 2017 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2018. Retrieved 16 September 2020.