Approved treatments for Gaucher disease

Overview

Treatment for Gaucher disease usually includes therapies that address the underlying cause of the disease, as well as supportive treatments to manage symptoms. The two main treatment approaches are enzyme replacement therapy and substrate reduction therapy. While these therapies cannot cure Gaucher disease, they can help manage symptoms, prevent complications, and improve quality of life for many people with the disease.

Enzyme replacement therapy

Enzyme replacement therapy (ERT) is a first-line treatment for many people with Gaucher disease. It works by replacing the missing glucocerebrosidase enzyme, helping the body break down the fatty substance that builds up in Gaucher disease.

Cerezyme (imiglucerase)

Cerezyme (imiglucerase) is an approved enzyme replacement therapy used to manage non-neurological symptoms in adults and children with Gaucher disease types 1 and 3. It is delivered via intravenous infusions at a dose and frequency that vary based on disease severity.

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VPRIV (Velaglucerase alpha)

VPRIV (velaglucerase alfa) is an approved enzyme replacement therapy designed to ease signs and symptoms of Gaucher disease type 1 in pediatric and adult patients. It is delivered via intravenous infusions, at a dose that is dependent on prior treatment history and therapeutic goals.

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Elelyso (taliglucerase alpha)

Elelyso (taliglucerase alfa) is an approved enzyme replacement therapy that is used to help manage certain disease complications and ease symptoms in adults and children, ages 4 and older, with Gaucher disease type 1. It is administered via intravenous infusions every other week.

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Substrate reduction therapy

Substrate reduction therapy (SRT) works by reducing the production of the fatty substance that accumulates in Gaucher disease. Taken as oral capsules, SRT may be an alternative to ERT for certain adults with Gaucher disease type 1.

Cerdelga

Cerdelga (eliglustat) is an approved substrate reduction therapy used to manage certain complications and alleviate symptoms of Gaucher disease type 1 in adults with a certain metabolic profile. It is taken once or twice daily as oral capsules

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Zavesca (miglustat)

Zavesca (miglustat) is an oral substrate reduction therapy approved for adults with mild to moderate Gaucher disease type 1 who cannot receive enzyme replacement therapy. Taken as oral capsules three times per day, it is designed to ease symptoms and prevent certain Gaucher disease complications.

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