What is Mepsevii used to treat?
Mepsevii (vestronidase alfa-vjbk) is used to treat pediatric and adult patients with an inherited lysosomal storage disorder called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.
What is mucopolysaccharidosis type VII (MPS VII)?
MPS VII is a very rare, genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulphate, chondroitin sulphate and heparan sulphate. Glycosaminoglycans (formerly called mucopolysaccharides) are complex polysaccharides that have a range of important biological roles. The inability to properly break down glycosaminoglycans leads to an accumulation of GAGs in the tissues, which can cause extensive tissue and organ damage. Symptoms of MPS VII can include an abnormally coarsened face, lung disease, heart valve abnormalities, enlarged liver and spleen, joint stiffness, short stature, cognitive impairment, and skeletal abnormalities.
The life expectancy of individuals with MPS VII depends on the severity of symptoms. Heart disease and airway obstruction are the major causes of death in MPS VII patients.
How does Mepsevii work to treat MPS VII?
Mepsevii contains vestronidase alfa, which is a recombinant form of human beta-glucuronidase used to correct the beta-glucuronidase deficiency.
Mepsevii is administered as an intravenous infusion every two weeks.
Common side effects include infusion site reactions, diarrhea, rash and anaphylaxis.
- Mepsevii Information for Consumers
- Mepsevii Information for Healthcare Professionals (includes dosage details)
- Side Effects of Mepsevii (detailed)