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IMPORTANT SAFETY INFORMATION: Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients... View more

How Cerezyme Helps

Cerezyme is the ONLY ERT (enzyme replacement therapy) that has shown long-term efficacy and safety in multiple studies over 10 years and has been prescribed for over 20 years.

View Indications and Usage

How Cerezyme may help

Cerezyme has helped adults and children with Gaucher disease type 1 by improving key disease symptoms over the long term, including:

Organ problems

Organ Problems

  • Enlarged spleen (splenomegaly) and liver (hepatomegaly)
Blood problems

Blood Problems

  • Low red blood cell count
  • Reduced platelets in the blood
Bone problems

Bone Problems

  • Low bone mineral density (BMD)
  • Bone pain
  • Bone crisis

Bone mineral density, or bone mass, is the amount of minerals in your bones.
Bone crisis is an episode of severe bone pain that lasts for more than 3 days and may be accompanied with other symptoms, such as fever.

Learn more about the signs and symptoms of Gaucher disease type 1

The studies behind Cerezyme

Cerezyme has been studied in adults and children who have Gaucher disease type 1.

  • Cerezyme was evaluated in prospective clinical trials to show its efficacy and safety.
  • Gaucher Registry studies retrospectively support the clinical trial efficacy and safety findings.

What is the Gaucher Registry?
The Gaucher Registry is an international database sponsored by Sanofi Genzyme that was initiated to keep track of the experiences of people with Gaucher disease. Data from the Registry is used to help researchers and physicians understand the impact of Gaucher disease and the effectiveness of long-term treatment. Since 1991, the Registry has collected voluntary information from over 6,000 people with Gaucher disease worldwide.

Key efficacy and safety studies

About the study

30 people with Gaucher disease type 1, aged 12 to 69 years old... Read more...

changes in spleen size compared to the start of the study

Enlarged spleen
6 months
  • 37.3% average decrease in spleen volume
    for Cerezyme
    (out of 8 patients)
  • 32.1% average decrease in spleen volume
    for alglucerase
    (out of 16 patients)
9 months
  • 47.1% average decrease in spleen volume
    for Cerezyme
    (out of 8 patients)
  • 42.2% average decrease in spleen volume
    for alglucerase
    (out of 16 patients)

changes in liver size compared to the start of the study

Enlarged liver
6 months
  • 13.4% average decrease in liver volume
    for Cerezyme
    (out of 8 patients)
  • 11.4% average decrease in liver volume
    for alglucerase
    (out of 16 patients)
9 months
  • 21.4% average decrease in liver volume
    for Cerezyme
    (out of 8 patients)
  • 16.4% average decrease in liver volume
    for alglucerase
    (out of 16 patients)

Cerezyme showed similar reductions to alglucerase in the size of enlarged organs in Gaucher disease type 1

Improved red blood cell levels compared to the start of the study

Blood and lung problems
6 months
  • 1.82 g/dL average increase in red blood
    cell level for Cerezyme
    (out of 15 patients)
  • 1.60 g/dL average increase in red blood
    cell level for alglucerase
    (out of 15 patients)
9 months
  • 2.54 g/dL average increase in red blood
    cell level for Cerezyme
    (out of 15 patients)
  • 2.28 g/dL average increase in red blood
    cell level for alglucerase
    (out of 15 patients)

Improved platelet count compared to the start of the study

Blood and lung problems
6 months
  • 21.5% average increase in platelet count for
    Cerezyme
    (out of 15 patients)
  • 33.5% average increase in platelet count for
    alglucerase
    (out of 15 patients)
9 months
  • 43.5% average increase in platelet count for
    Cerezyme
    (out of 15 patients)
  • 53.2% average increase in platelet count for
    alglucerase
    (out of 15 patients)

Cerezyme showed similar improvements as alglucerase in blood problems in Gaucher disease type 1, including decreased red blood cell level and platelet count

This study established the efficacy and safety of Cerezyme in certain bone parameters

About the study

This study included 33 children and adults with Gaucher disease type 1 who reported at least 1 bone problem, such as bone crisis, death of cells in the bones or joints, bone tissue destruction, and fractures. Read more...

Cerezyme reduced bone pain occurrence over 48 months

Start of study
24 out of 33 patients (73%) reported bone pain
After 48 months
9 out of 23 patients (39%) reported bone pain

Cerezyme reduced bone crisis occurrence over 48 months

Start of study
13 out of 33 patients (39%) had a history of bone crises
The other 20 patients had no history of bone crises
After 48 months
2 out of those 13 patients (15%) experienced a bone crisis during the study
One patient with no bone crisis history experienced a crisis during the study

After 48 months, patients achieved near-normal lumbar spine bone mineral density (BMD)

Bone pain was assessed by patient report on a scale: none, very mild, mild, moderate, severe, or extreme. Bone crisis is severe bone pain lasting several days, often accompanied by fever. Bone mineral density (BMD) is a measure of bone health and risk for fracture. Low BMD means weakened bones.

Long-term study

About the study

This was an observational study where researchers reviewed information that had been collected in the Gaucher Registry for 757 children and adults with Gaucher disease type 1... Read more...

Cerezyme showed improvements in organ, blood, and bone problems in Gaucher disease type 1 after 10 years of treatment. Compared to the start of treatment:

Cerezyme reduced the size of enlarged organs

Enlarged spleen
  • 73% reduction in average spleen volume
    (out of 107 patients)
Enlarged liver
  • 44% reduction in average liver volume
    (out of 105 patients)

Cerezyme improved blood problems

Blood and lung problems
  • 2.4 g/dL increase in average red blood cell level
    (out of 376 patients)
Blood and lung problems
  • 75% increase in average platelet count
    (out of 397 patients)

Cerezyme reduced bone problems

Bone problems
  • 57% reduction in the number of patients reporting bone pain
    • 98 out of 187 patients had reported a history of bone pain before treatment
    • 42 of these 98 patients reported bone pain 10 years after first infusion
Bone problems
  • 93% reduction in the number of patients reporting bone crisis
    • 27 out of 169 patients had reported a history of bone crisis before treatment
    • 2 of these 27 patients reported bone crisis 10 years after first infusion

Bone crisis is defined as an episode of severe bone pain that lasts for more than 3 days that requires pain relief, restricted movement, and might be seen with other symptoms including fever.

Long-term treatment with Cerezyme reduced organ, blood, and bone problems in patients with Gaucher disease type 1

Bone studies

This is one of 2 registry studies that supported the findings of the 48-month bone trial

About the study

In this 8-year observational study, researchers reviewed data from adult patients enrolled in the Gaucher Registry who had a history of lumbar spine BMD measurements recorded

  • Up to 8 years of BMD measurements were analyzed for 160 patients who received no treatment and for 342 patients treated with Cerezyme

Cerezyme improved and normalized spine BMD with long-term treatment

Spine BMD improvement

Improvement within 12 months

  • Cerezyme showed a trend toward lumbar spine BMD improvement

Continued long-term effectiveness

  • Patients achieved near-normal lumbar spine BMD after 8 years

Bone mineral density (BMD) is a measure of mineral in bones and bone strength. Low BMD means weakened bones.

This is one of 2 registry studies that supported the findings of the 48-month bone trial

About the study

In this 4-year observational study, researchers reviewed data from adult and pediatric patients enrolled in the Gaucher Registry who had bone crisis and/or bone pain information for 1 year before treatment, and for each of 3 years after the start of Cerezyme. The year before treatment was used for comparison. Read more...

Cerezyme reduced bone pain and bone crises

Patients experiencing bone pain
Before treatment 49% (119 out of 244 patients)
After 1 year of ERT 30% (74 out of 244 patients)
After 3 years of ERT 30% (73 out of 244 patients)
Patients experiencing bone crises
Before treatment 17% (38 out of 219 patients)
After 1 year of ERT 4.6% (10 out of 219 patients)
After 3 years of ERT 2.7% (6 out of 219 patients)

Bone crisis is severe bone pain lasting several days, often accompanied by fever.

Pediatric study

About the study

This was an observational study where researchers reviewed data from 884 pediatric patients with Gaucher disease type 1 who were enrolled in the Gaucher Registry. The study patients had intact spleens and were receiving alglucerase (22.4%) or Cerezyme (77.6%). Read more...

Long-term Cerezyme treatment for children with Gaucher disease type 1 improved organ, blood, and low BMD problems

Cerezyme reduced the size of enlarged organs over 8 years

Enlarged spleen
  • 79% reduction in median spleen volume
    (out of 458 patients)
    • Half the treatment effect was seen within 1 year of treatment
Enlarged liver
  • 45% reduction in median liver volume
    (out of 420 patients)
    • Half the treatment effect was seen within 1 year of treatment

Cerezyme improved blood problems over 8 years

Blood problems
  • 2 g/dL increase in median red blood cell levels
    (out of 771 patients)
    • 0% of children had anemia after 6 years
    • Red blood cell levels increased to normal levels after 8 years
Blood problems
  • 74% increase in median platelet count
    (out of 768 patients)

Cerezyme increased bone mineral density (BMD) over 12 years

Bone problems

Long-term use of Cerezyme improved BMD in children

What is Cerezyme?

What is Cerezyme?

An ERT that can help patients with Gaucher disease

Learn more
Signs and symptoms 

Why treat Gaucher?

Gaucher disease symptoms may lead to serious complications

See details

Indication & Usage

Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

  • anemia (low red blood cell count)
  • thrombocytopenia (low blood platelet count)
  • bone disease
  • hepatomegaly or splenomegaly (enlarged liver or spleen)

Important Safety Information

Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.

High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.

Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.

Please see Full Prescribing Information (PDF).