Cipaglucosidase alfa

Cipaglucosidase alfa is a prescription medicine used to treat Gaucher disease type 1, an inherited disorder caused by a deficiency of the enzyme glucocerebrosidase. It works by replacing the missing enzyme in the body to help break down certain fatty substances in the body. Cipaglucosidase alfa is given by intravenous injection. Common side effects may include diarrhea, headache, nausea, abdominal pain, and injection site reactions.

Cipaglucosidase alfa, sold under the brand name Pombiliti, is a medication used in combination with miglustat for the treatment of late-onset Pompe disease (acid α-glucosidase [GAA] deficiency) in adults. Here's a breakdown of its key aspects:

Function:

• Cipaglucosidase alfa belongs to a class of drugs called enzyme replacement therapy (ERT).
• In individuals with Pompe disease, the body lacks a functional enzyme called acid α-glucosidase (GAA), leading to the buildup of glycogen (a complex sugar) in various tissues.
• Cipaglucosidase alfa acts as a replacement for the missing GAA enzyme, helping break down glycogen and potentially alleviate symptoms associated with the disease.