Imiglucerase

Gaucher disease is characterized by a deficiency of β-glucocerebrosidase activity, which results in accumulation of glucocerebroside in various tissues including liver, spleen, and bone marrow. The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages.

• CEREZYME is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients. CEREZYME is a hydrolytic lysosomal glucocerebrosidase-specific enzyme indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients.

• None. None.contraindicated

Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) The recommended dosage is 2.5 units/kg three times a week to 60 units/kg once every two weeks administered intravenously. ( 2.2 ) Titrate the dosage based on disease severity and therapeutic goals for the patient. ( 2.2 ) See the full prescribing information for dosage modifications due to hypersensitivity reactions and/or IARs. ( 2.3 ) See the full prescribing information for preparation and administration instructions. ( 2.4 , 2.5 ) 2.1 Recommendations Prior to CEREZYME Treatment Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . For patients who experience hypersensitivity reactions to CEREZYME, premedicate with antihistamines and/or corticosteroids. Monitor patients for the occurrence of new hypersensitivity reactions [see Warnings and Precautions (5.1) ] . 2.2 Recommended Dosage and Administration Ensure physicians knowledgeable in the management of patients with Gaucher disease direct therapy with CEREZYME. The recommended dosage of CEREZYME is 2.5 units/kg three times a week to 60 units/kg once every two weeks administered intravenously.

Infusion-Associated Reactions (IARs) : If a severe IAR occurs, discontinue CEREZYME immediately and initiate appropriate medical treatment. ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies, including CEREZYME. In addition, other hypersensitivity reactions have included pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, cough, cyanosis, tachycardia, and hypotension [see Adverse Reactions (6.1) ] . Patients with antibody to imiglucerase have a higher risk of hypersensitivity reactions. Conversely, not all patients with symptoms of hypersensitivity have detectable IgG antibody. Consider periodic monitoring of patients during the first year of treatment for IgG antibody formation [see Adverse Reactions (6.2) ] . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine.

The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions [see Warnings and Precautions (5.2) ] Adverse reactions reported in adults and pediatric patients include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-633-1610 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials and Postmarketing Experience The following adverse reactions associated with the use of imiglucerase were identified in clinical studies or postmarketing reports. Because some of these reactions were reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. See Table 2 for adverse reactions occurring in adults and pediatric patients treated with CEREZYME in clinical trials and the postmarketing setting. Table 2: Adverse Reactions in Adults and Pediatric Patients Treated with CEREZYME Adverse Reactions Nervous system disorders dizziness, headache Cardiac disorders tachycardia Vascular disorders cyanosis, Signs and symptoms suggestive of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] and other infusion-associated reactions [see Warnings and Precautions (5.2) ] .

8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to CEREZYME during pregnancy. Pregnant women exposed to CEREZYME and health care providers are encouraged to contact the Gaucher patient registry at 1-800-745-4447, extension 15500 or visit www.registrynxt.com. Risk Summary Available data on more than 500 pregnancies from the international Gaucher Disease registry, postmarketing reports, published observational studies and case reports with CEREZYME or non–US-licensed imiglucerase use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks associated with symptomatic Type I Gaucher disease in pregnancy (see Clinical Considerations ) . No animal reproduction studies have been conducted with imiglucerase. The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

During one-hour intravenous infusions of four doses (7.5, 15, 30, 60 units/kg) of CEREZYME, steady-state enzymatic activity was achieved by 30 minutes. Following infusion, the half-life of plasma enzymatic activity ranged from 3.6 to 10.4 minutes.

• May 23, 1994BLABLA020367Genzyme

1. 1Pyrexia2124.6%
2. 2Fatigue1803.9%
3. 3Death1723.7%
4. 4Pneumonia1723.7%
5. 5Fall1683.6%
6. 6Exposure During Pregnancy1633.5%
7. 7Weight Increased1493.2%
8. 8Dyspnoea1483.2%
9. 9Bone Pain1463.2%
10. 10Malaise1362.9%
11. 11Weight Decreased1292.8%
12. 12Arthralgia1272.7%
13. 13Disease Progression1262.7%
14. 14Pain1162.5%
15. 15Condition Aggravated1152.5%

The 10 most recently updated of 71 ClinicalTrials.gov registrations naming Imiglucerase as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Genetic Studies of Lysosomal Storage Disorders
  Enrolling by invitation · Observational · 1,000 enrolled · National Human Genome Research Institute (NHGRI)
  NCT00001215updated 2026-06-12
• Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
  Active not recruiting · Phase 2 · Interventional · 12 enrolled · Genzyme, a Sanofi Company
  NCT02843035updated 2026-06-11
• Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
  Completed · Observational · 64 enrolled · National Human Genome Research Institute (NHGRI)
  NCT00302146updated 2026-06-05
• Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD
  Active not recruiting · Phase 2 · Interventional · 237 enrolled · Bial R&D Investments, S.A.
  NCT05819359updated 2026-05-13
• International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
  Recruiting · Observational · 12,000 enrolled · Genzyme, a Sanofi Company
  NCT00358943updated 2026-04-14
• Genotypic Influences on Network Progression in Parkinson's Disease
  Active not recruiting · Observational · 32 enrolled · Northwell Health
  NCT04228172updated 2026-04-13
• Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 7 enrolled · Prevail Therapeutics
  NCT04411654updated 2026-04-01
• Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease
  Completed · Interventional · 4 enrolled · InSightec
  NCT04370665updated 2026-03-20
• Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 32 enrolled · Prevail Therapeutics
  NCT04127578updated 2026-03-13
• MR-guided Focused Ultrasound Plus GCase
  Recruiting · Interventional · 14 enrolled · InSightec
  NCT05565443updated 2026-01-23

Frequently asked questions

How does Imiglucerase work?

Gaucher disease is characterized by a deficiency of β-glucocerebrosidase activity, which results in accumulation of glucocerebroside in various tissues including liver, spleen, and bone marrow. The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages.

What is Imiglucerase used for?

According to FDA labeling, Imiglucerase carries indications including: CEREZYME is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients. CEREZYME is a hydrolytic lysosomal glucocerebrosidase-specific enzyme indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Imiglucerase?

Imiglucerase is classified as Enzymes, Hydrolytic Lysosomal Glucocerebroside-specific Enzyme, Enzymatic Activity, Lipid Metabolism Alteration.

What are the brand names for Imiglucerase?

Imiglucerase is marketed under brand names including Cerezyme.

What are the contraindications for Imiglucerase?

Imiglucerase labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.