Vestronidase alfa

Vestronidase alfa (also known as Mepsevii) is a recombinant form of the human enzyme beta-glucuronidase. It is used as an enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.

Mechanism of Action

Vestronidase alfa works by replacing the deficient beta-glucuronidase enzyme in patients with MPS VII. This enzyme is responsible for the breakdown of glycosaminoglycans (GAGs), complex molecules that are part of many tissues in the body. In MPS VII, a deficiency in beta-glucuronidase leads to an accumulation of GAGs in various tissues and organs, causing a range of symptoms.

Clinical Use

Vestronidase alfa is administered via intravenous infusion and is typically given every two weeks. The dosage is based on the patient's body weight. The most common side effects include diarrhea, rash, and infusion-related reactions.

Development and Approval

Vestronidase alfa was developed by Ultragenyx Pharmaceutical and was approved by the Food and Drug Administration (FDA) in 2017. It is the first and only FDA-approved treatment for MPS VII.

See Also

• Enzyme replacement therapy
• Mucopolysaccharidosis
• Beta-glucuronidase
• Ultragenyx Pharmaceutical