Importance of Early Diagnosis

Type 1 Gaucher disease is a progressive, often debilitating, multisystemic disorder. While Cerezyme has been shown to improve many of the signs and symptoms of Type 1 Gaucher disease,¹ some debilitating and disabling manifestations may become irreversible if treatment is delayed. For instance, it has been observed in Registry data that bone pain and bone crises improved with treatment.¹ However, advanced liver disease may become irreversible; advanced anemia and thrombocytopenia may show some improvement, but often do not normalize.

In addition, some manifestations of Gaucher disease respond more slowly to treatment than others. For instance, it has been observed in Registry data that increases in bone mineral density occurred more slowly than hematological or visceral improvements.³ Near normal BMD was seen after two years in children⁴ and eight years in adults.³

Recognizing Early Symptoms

One reason diagnosis and treatment may be delayed is that some manifestations of Gaucher disease are similar to those of other, more common diseases, so patients may be misdiagnosed. For example, joint pain may be attributed to arthritis or growing pains. Low levels of red blood cells or platelets may at first be diagnosed as a blood disorder. Other misdiagnoses may include:⁵

• Leukemia
• Lymphoma
• Bleeding disorders
• Osteomyelitis

Clusters of signs and symptoms in two or more organ systems often suggest an underlying genetic disorder.

If you suspect Gaucher disease, early diagnosis and treatment are critical to ensure optimal outcomes. Please see Diagnosing Gaucher Disease for more information.

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:

1. anemia
2. thrombocytopenia
3. bone disease
4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies, and these patients have a higher risk of hypersensitivity reaction. Therefore periodic monitoring is suggested; caution should be exercised in patients with antibodies or prior symptoms of hypersensitivity. Symptoms suggestive of hypersensitivity occurred in 6.6% of patients, and include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension.

Reactions related to Cerezyme administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported adverse events include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Adverse events associated with the route of administration include discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of injection.

To report suspected adverse reactions, contact Genzyme at 800-745-4447, option 2 or FDA at 800-FDA-1088 or http://www.fda.gov/Safety/MedWatch

Please see Full Prescribing Information (PDF).