Vestronidase alfa

Vestronidase alfa
Names
Trade namesMepsevii
Other namesVestronidase alfa-vjbk
Clinical data
Drug classEnzyme[1]
Main usesMucopolysaccharidosis type VII (Sly syndrome)[2]
Side effectsDiarrhea, rash, anaphylaxis[1][2]
WHO AWaReUnlinkedWikibase error: ⧼unlinkedwikibase-error-statements-entity-not-set⧽
Routes of
use		Injection
Typical dose4 mg/kg[2]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status
  • US: ℞-only
  • EU: Rx-only
  • In general: ℞ (Prescription only)
Chemical and physical data
FormulaC3308H4996N874O940S16
Molar mass72562.49 g·mol−1

Vestronidase alfa, sold under brand name Mepsevii, is a medication used to treat mucopolysaccharidosis type VII (Sly syndrome).[2] It is given by injection into a vein over four hours.[2]

Common side effects include swelling at the site of injection, diarrhea, rash, and anaphylaxis.[1][2] It is a recombinant form of the human enzyme beta-glucuronidase; and works by replacing this missing enzyme.[2][1]

Vestronidase alfa was approved in the United States in 2017 and Europe in 2018.[1][2] 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.[3]

Medical uses

Mepsevii is indicated for the treatment of non-neurological manifestations of mucopolysaccharidosis VII (MPS VII; Sly syndrome).[2][4]

Dosage

The typical dose is 4 mg/kg every two weeks.[2]

History

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] Participants received treatment with vestronidase alfa at doses up to 4 mg/kg once every two weeks for up to 164 weeks.[5] Efficacy was primarily assessed via the six-minute walk test in ten participants who could perform the test.[5] After 24 weeks of treatment, the mean difference in distance walked relative to placebo was 18 meters.[5] Additional follow-up for up to 120 weeks suggested continued improvement in three participants and stabilization in the others.[5] Two participants in the vestronidase alfa development program experienced marked improvement in pulmonary function.[5] Overall, the results observed would not have been anticipated in the absence of treatment.[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. The trial was conducted at four sites in the United States.[6] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]

The benefit and side effects of vestronidase alfa were based primarily on one trial.[6] Participants were randomly assigned to four groups.[6] Three groups of participants received placebo treatment before starting vestronidase alfa treatment and one group received vestronidase alfa only.[6] vestronidase alfa or placebo were given once every two weeks as intravenous (IV) infusions.[6] Neither participants nor healthcare providers knew which treatment was given until after the trial was competed.[6]

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.[6] The 6MWT measured the distance a patient could walk on a flat surface in 6 minutes.[6] An additional follow-up using 6MWT was done for up to 120 weeks.[6]

The application for vestronidase alfa was granted fast track designation, orphan drug designation, and a rare pediatric disease priority review voucher.[5] This was the twelfth rare pediatric disease priority review voucher issued.[5]

The U.S. 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.[5]

References

  1. 1 2 3 4 5 "Vestronidase Alfa-vjbk Monograph for Professionals". Drugs.com. Archived from the original on 15 September 2021. Retrieved 13 September 2021.
  2. 1 2 3 4 5 6 7 8 9 10 "Mepsevii EPAR". European Medicines Agency (EMA). Archived from the original on 8 April 2020. Retrieved 28 February 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  3. ↑ "Mepsevii Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 15 September 2021. Retrieved 13 September 2021.
  4. ↑ "Mepsevii- vestronidase alfa injection". DailyMed. 19 November 2017. Archived from the original on 21 January 2021. Retrieved 5 August 2020.
  5. 1 2 3 4 5 6 7 8 9 10 11 12 "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. 1 2 3 4 5 6 7 8 9 "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. Archived from the original on 23 October 2020. Retrieved 16 September 2020.
Identifiers:
  • "Vestronidase alfa". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on 9 July 2021. Retrieved 1 July 2021.
  • 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
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