Cerezyme becomes 1st FDA-approved therapy for type 3 Gaucher
Approval expanded to non-neurological symptoms in type 3 children, adults
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Note: This story was updated Jan. 20, 2026, to clarify that the label update also removed age restrictions, allowing Cerezyme to be used from birth.
The U.S. Food and Drug Administration (FDA) has expanded the approval of Cerezyme (imiglucerase) to include the treatment of non-neurological symptoms in children and adults with Gaucher disease type 3.
The decision makes Sanofi‘s Cerezyme the first therapy approved in the U.S. to treat type 3 Gaucher. The treatment, an enzyme replacement therapy (ERT), has been approved for this indication in Europe since 1997.
The FDA also updated Cerezyme’s label for type 1 Gaucher disease, a condition for which the therapy has been approved since 1994. Under the revised label, Cerezyme is approved to treat non-central nervous system manifestations of both type 1 and type 3 Gaucher disease in all adults and children. Its use in type 1 disease had been limited to patients ages 2 and older with one or more specific complications, such as anemia (low red blood cell counts), low platelet levels, bone disease, or liver or spleen enlargement.
Gaucher disease is caused by mutations that result in a missing or defective glucocerebrosidase (GCase) enzyme, which is needed to break down certain fatty molecules so the body can reuse them. Without sufficient GCase, these fatty molecules accumulate to harmful levels in certain cells and tissues, driving Gaucher symptoms.
Gaucher is typically characterized by symptoms including enlarged organs, blood abnormalities, and bone disease, but some patients can also experience neurological complications. Type 1 Gaucher disease, the most common form, does not cause neurological symptoms. Type 3, a rarer form of the disease, is marked by slowly progressing neurological symptoms, and also causes many of the organ symptoms seen in type 1.
Expanded approval comes after registry analysis
Cerezyme works by supplying the body with a functional version of GCase, helping reduce the toxic buildup of fatty molecules. Its original approval for type 1 disease was largely based on a Phase 3 clinical trial showing improvements in anemia, platelet counts, and liver and spleen volume.
The label expansion was supported by data from the International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry (NCT00358943), a Sanofi-sponsored registry that includes information from thousands of Gaucher patients worldwide.
The analysis included more than 1,000 people with type 1 disease and more than 100 with type 3 who received Cerezyme as a first-line treatment and were followed for about two years. Results showed that hemoglobin (the protein that carries oxygen in red blood cells) and platelet levels increased over time in both groups.
Patients with Gaucher type 3 also showed reductions in liver and spleen volume, along with improvements in growth measures.
Cerezyme is delivered by intravenous infusion at a recommended dose ranging from 2.5 units/kg of body weight, administered three times a week, to 60 units/kg given once every two weeks. Dosing and scheduling are tailored to disease severity and individual treatment goals.
The therapy carries a boxed warning noting it can cause severe allergic reactions, including anaphylaxis. For this reason, it should be administered under the supervision of a healthcare provider who knows how to manage these reactions, and in a setting where monitoring equipment and support measures are available.
