MEPSEVII (vestronidase alfa-vjbk) · ULTRAGENYX PHARM INC
Mepsevii (vestronidase alfa-vjbk) is a recombinant human lysosomal beta glucuronidase indicated for the treatment of Mucopolysaccharidosis VII, also known as Sly syndrome. The therapy is approved for use in both pediatric and adult patients to address the underlying enzyme deficiency associated with the disorder. However, the effect of this treatment on the central nervous system manifestations of the disease has not been determined.
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.
Details
- Status
- Prescription
- First Approved
- 2017-11-15
- Patent Cliff
- 2024
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
MEPSEVII Approval History
What MEPSEVII Treats
1 indicationsMEPSEVII is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Mucopolysaccharidosis VII
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 ( ...
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 ( 2.2 ), Warnings and Precautions ( 5.1 )]. WARNING: ANAPHYLAXIS See full prescribing information for complete boxed warning. Anaphylaxis has occurred with MEPSEVII administration, as early as the first dose ( 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 ( 2.2 , 5.1 ). Immediately discontinue the MEPSEVII infusion if the patient experiences anaphylaxis ( 2.2 , 5.1 ).
ULTRAGENYX PHARM INC's other novel FDA approvals
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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 in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Limitations of Use The effect of MEPSEVII on the central nervous system manifestations of MPS VII has not been determined. MEPSEVII is a recombinant human lysosomal beta glucuronidase indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Limitations of Use The effect of MEPSEVII on the central nervous system manifestations of MPS VII has not been determined.
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.