Cerezyme is the ONLY ERT (enzyme replacement therapy) that has shown long-term efficacy and safety in multiple studies over 20 years and has been prescribed for over 25 years.
Cerezyme has helped adults and children with Gaucher disease type 1 by improving key disease symptoms over the long term, including:
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
Cerezyme has been studied in adults and children who have Gaucher disease type 1.
What is the Gaucher Registry?
The Gaucher Registry is an international database sponsored by Sanofi 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.
30 people with Gaucher disease type 1, aged 12 to 69 years old
The 6-month pivotal clinical trial compared Cerezyme to alglucerase to confirm that both worked similarly to relieve certain disease symptoms, including organ and blood problems, in people with Gaucher disease type 1.*
Alglucerase was the first ERT developed by Genzyme for people with Gaucher disease type 1. Alglucerase is no longer available and was replaced by Cerezyme.
changes in spleen size compared with the start of the study
changes in liver size compared with the start of the study
Improved red blood cell levels compared with the start of the study
Improved platelet count compared with the start of the study
Cerezyme showed similar improvements as alglucerase in blood problems in Gaucher disease type 1, including decreased red blood cell level and platelet count
BONE IMPROVEMENTS
Bone x-rays showed improvements in cortical thickness and lucencies in 7 of 11 Cerezyme-treated patients in the initial analysis period (6 months).
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, and received Cerezyme every 2 weeks for up to 48 months.
The study measured bone response, as shown by bone pain, bone crisis, and lumbar spine and femur (thigh bone) BMD.
| Start of study |
| 24 out of 33 patients (73%) reported bone pain |
| After 48 months |
| 9 out of 23 patients (39%) reported bone pain |
| 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 |
Bone pain was assessed by patient report on a scale: none, very mild, mild, moderate, severe, or extreme. Bone crisis was defined as pain with acute onset requiring immobilization of the affected area, narcotics for pain relief, and may be accompanied by 1 or more of the following: periosteal elevation, elevated white blood cells, fever, or debilitation of >3 days. Bone mineral density (BMD) is a measure of bone health and risk for fracture. Low BMD means weakened bones.
This was an observational study where researchers reviewed information that had been collected in the Gaucher Registry for 475 children and adults with Gaucher disease type 1 who were treated with Cerezyme for about 20 years.
Before switching to Cerezyme, some patients in the study received alglucerase—the first ERT developed by Genzyme for people with Gaucher disease type 1.
Cerezyme showed improvements in organ, blood, and bone problems in Gaucher disease type 1 patients over 20 years of treatment. Compared to the start of treatment:
The following data is for Cerezyme-treated patients with intact spleens.
Cerezyme reduced the size of enlarged organs
Cerezyme improved blood problems
Cerezyme reduced bone problems
The above values improved in patients without spleens as well (except for the spleen volume).
Bone crisis is described as “pain with acute onset which requires immobilization of the affected area, narcotics for the relief of pain, and may be accompanied by 1 or more of the following: periosteal elevation, elevated white blood cell count, fever or debilitation of >3 days” since the last assessment.
The study found that long-term treatment with Cerezyme reduced organ, blood, and bone problems in patients with Gaucher disease type 1.
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
Cerezyme impact on spine BMD with long-term treatment
Bone mineral density (BMD) is a measure of mineral in bones and bone strength. Low BMD means weakened bones.
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.
Cerezyme effect on 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 crises are often associated with acute bone infarction. They typically begin with regional dull, aching pains that become intense and excruciating over about 3 days.
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%).
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
Cerezyme improved blood problems over 8 years
Cerezyme EFFECT ON bone mineral density (BMD) over 12 years
Long-term use of Cerezyme showed an impact on BMD in children
Gaucher disease type 1 symptoms may lead to serious complications
See detailsCerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:
Hypersensitivity and Infusion-Associated Reactions: Serious allergic reactions (anaphylaxis) have been reported in patients treated with Cerezyme. Symptoms suggestive of an allergic reaction have been reported during or shortly after an infusion and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), rapid heart rate, and low blood pressure. Inform your doctor and seek medical care if you experience any of these symptoms. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.
Immune Responses: 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.
Adverse Reactions:
Adverse reactions reported in adults include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting.
Adverse reactions reported in pediatric patients 2 years of age and older are similar to adults.
Please see the Full Prescribing Information.
To report SUSPECTED ADVERSE REACTIONS, contact Sanofi Medical Information at 1-800-745-4447, Option 2.