TheraRadar
Data updated: May 26, 2026

MEPSEVII (vestronidase alfa-vjbk)

First-in-Class Orphan Drug Priority Review Fast Track
Rare Disease Approved 2017-11-15

MEPSEVII is indicated for the treatment of Mucopolysaccharidosis VII.

Source: FDA Label • ULTRAGENYX PHARM INC • Lysosomal beta Glucuronidase

How MEPSEVII Works

Mucopolysaccharidosis VII is caused by a deficiency of the enzyme beta glucuronidase (GUS), which results in the accumulation of glycosaminoglycans (GAGs) and subsequent multisystem tissue damage. Vestronidase alfa-vjbk provides an exogenous source of the GUS enzyme that is internalized into cellular lysosomes via mannose-6-phosphate receptors on the cell surface. Once inside the lysosomes, the enzyme facilitates the catabolism of accumulated GAGs in affected tissues.

2
Indications
--
Phase 3 Trials
1
Priority Reviews
8
Years on Market

Details

Status
Prescription
First Approved
2017-11-15
Patent Cliff
2024

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Routes
INJECTION
Dosage Forms
INJECTABLE

Companies

Active Ingredient: VESTRONIDASE ALFA-VJBK

MEPSEVII Approval History

2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
3 FDA actions from 2017 to 2020 · 1 indication expansions
Dec 2020 SUPPL
Efficacy
Dec 2019 SUPPL
Label · Labeling
Nov 2017 ORIGINAL Priority
New Drug · Type 1 - New Molecular Entity

What MEPSEVII Treats

1 indications

MEPSEVII is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Mucopolysaccharidosis VII
Source: FDA Label

MEPSEVII Boxed Warning

ANAPHYLAXIS Anaphylaxis has occurred with MEPSEVII administration, as early as the first dose [see Warnings and Precautions ( 5.1 )] , therefore appropriate medical support should be readily available when MEPSEVII is administered. Closely observe patients during and for 60 minutes after MEPSEVII infusion [see Dosage and Administration ( 2.2 ), Warnings and Precautions ( 5.1 )]. Immediately discontinue the MEPSEVII infusion if the patient experiences anaphylaxis [see Dosage and Administration ( ...

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Active Pipeline

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Key Completed Trials

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Trial Timeline

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

MEPSEVII FDA Label Details

Indications & Usage

FDA Label (PDF)

MEPSEVII is indicated for the treatment of Mucopolysaccharidosis VII.

⚠️ BOXED WARNING

WARNING: ANAPHYLAXIS Anaphylaxis has occurred with MEPSEVII administration, as early as the first dose [see Warnings and Precautions ( 5.1 )] , therefore appropriate medical support should be readily available when MEPSEVII is administered. Closely observe patients during and for 60 minutes after ME...

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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.