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Cerezyme®
Cerezyme is an enzyme replacement therapy that provides the missing glucocerebrosidase enzyme to break down glucocerebroside in patients with Gaucher disease.
Cerezyme is an enzyme replacement therapy that provides the missing glucocerebrosidase enzyme to break down glucocerebroside in patients with Gaucher disease. Used for Gaucher disease type 1 (non-neuronopathic form), Gaucher disease type 3 (chronic neuronopathic form).
At a glance
| Generic name | Cerezyme® |
|---|---|
| Sponsor | ISU Abxis Co., Ltd. |
| Drug class | Enzyme replacement therapy |
| Target | Glucocerebrosidase (β-glucosidase) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | FDA-approved |
Mechanism of action
Gaucher disease is a lysosomal storage disorder caused by deficiency of the enzyme glucocerebrosidase (also called β-glucosidase), leading to accumulation of glucocerebroside in macrophages throughout the body. Cerezyme is a recombinant form of this enzyme that is infused intravenously to replace the deficient enzyme and reduce substrate accumulation in affected tissues. This reduces organomegaly, anemia, thrombocytopenia, and bone disease associated with the disease.
Approved indications
- Gaucher disease type 1 (non-neuronopathic form)
- Gaucher disease type 3 (chronic neuronopathic form)
Common side effects
- Infusion reactions
- Fatigue
- Headache
- Nausea
- Diarrhea
- Antibody formation
Key clinical trials
- Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease (NA)
- Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (PHASE3)
- Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (PHASE4)
- Eliglustat on Gaucher Disease Type IIIB (NA)
- Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease (PHASE4)
- A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease (PHASE3)
- Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) (PHASE1, PHASE2)
- An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cerezyme® CI brief — competitive landscape report
- Cerezyme® updates RSS · CI watch RSS
- ISU Abxis Co., Ltd. portfolio CI