Cipaglucosidase alfa

Cipaglucosidase alfa is a enzyme replacement therapy used for the treatment of Mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. It is a recombinant form of the human enzyme beta-glucuronidase, which is deficient in individuals with MPS VII.

Mechanism of Action

Cipaglucosidase 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 individuals with MPS VII, the lack of beta-glucuronidase leads to an accumulation of GAGs in various tissues and organs, causing the symptoms of the disease. By providing a source of functional beta-glucuronidase, cipaglucosidase alfa helps to reduce the accumulation of GAGs and alleviate the symptoms of MPS VII.

Clinical Use

Cipaglucosidase alfa is administered via intravenous infusion and the dosage is based on the patient's body weight. The treatment is typically given every other week. The most common side effects include fever, vomiting, headache, nausea, and abdominal pain. Serious side effects can include allergic reactions and infusion-related reactions.

Development and Approval

Cipaglucosidase alfa was developed by Ultragenyx Pharmaceutical Inc. and was approved by the U.S. Food and Drug Administration (FDA) in 2017 under the brand name Mepsevii. It is the first and only treatment approved for MPS VII.

See Also

• Mucopolysaccharidosis
• Enzyme replacement therapy
• Beta-glucuronidase
• U.S. Food and Drug Administration

References

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