CAMBRIDGE, Mass., Sept. 9 /PRNewswire-FirstCall/ -- Genzyme General (Nasdaq: GENZ - News), a division of Genzyme Corp., today announced the publication of the most extensive set of data ever reported on the long- term effectiveness of enzyme replacement therapy with Cerezyme® (imiglucerase for injection) for patients with Type 1 Gaucher disease. The report, which appears in the American Journal of Medicine, describes the clinical success achieved by more than 1,000 patients worldwide followed in the Gaucher Registry who have received Cerezyme therapy for as long as five years. Data from the report were presented last week at the annual symposium of the Society for the Study of Inborn Errors of Metabolism in Dublin, Ireland.
The long-term findings described in the report build upon the results of earlier studies by showing that Cerezyme therapy reverses and prevents the progression of the major clinical manifestations of Gaucher disease. The authors concluded that patients achieved and maintained a complete or partial elimination of anemia and platelet deficiency and a reduction of the enlargement of the liver and spleen. Improvement or prevention of bone pain and bone crises was also observed. These clinical results, together with more than 11 years of safe and effective clinical use, have made Cerezyme therapy the standard of care for the treatment of individuals with Type 1 Gaucher disease.
"The data presented result from an unprecedented collaboration between the Gaucher medical and patient communities worldwide, aimed at better understanding the impact Cerezyme therapy has had on altering the natural history of this rare hereditary disease," said lead author Neal J. Weinreb, MD, University Hospital and Medical Center, Tamarac, Florida. "We anticipate that these results will provide a benchmark for the expectations and goals of therapy, and will help guide physicians in developing appropriate care plans for their patients with Gaucher disease."
Type 1 Gaucher disease is a chronic and debilitating disorder caused by a deficiency of the enzyme glucocerebrosidase. Patients with Type 1 Gaucher disease typically suffer from anemia, easy bleeding and bruising, enlargement of the spleen and liver, and deterioration of bones leading to frequent fractures and other complications.
The report shows sustained improvement in several bodily systems through five years of enzyme replacement therapy for patients with a wide range of clinical symptoms. "Despite the variability of disease severity and symptomatology in this large cohort of patients, the response to Cerezyme therapy was remarkably consistent," said Dr. Weinreb. Based on the results, the authors described the clinical outcomes that should be expected with long-term Cerezyme therapy. For example, approximately 90 percent of all patients with anemia should achieve normal levels of hemoglobin within two years of initiation of Cerezyme therapy. This response is sustained for up to five years among patients who had achieved normal levels, while those below normal continue to improve for up to five years.
Similarly, patients' low platelet counts should normalize within one year if their spleen had been previously removed, a procedure that is now rarely necessary since the availability of enzyme replacement therapy. In patients with an intact spleen, platelet counts should increase by 50 to 100 percent within 12 months, with further improvements through five years.
Liver sizes should decrease by 20-30 percent in one to two years and up to 40 percent in five years. Improvement in spleen size is even more pronounced, and should decrease by 30 percent in one year and 50 percent after two years.
Resolution of bone pain should occur in 50 percent of symptomatic patients within one to two years, while recurrent or new episodes of bone crises are substantially reduced, according to the report. The Gaucher Registry, sponsored by Genzyme and ongoing since enzyme replacement therapy was introduced in 1991, is the largest repository of information on Gaucher disease. It tracks the clinical outcomes of more than 2,500 Gaucher patients regardless of treatment status, and involves the active participation of approximately 250 physicians from 46 countries who make up the International Collaborative Gaucher Group. The Gaucher Registry is coordinated by a scientific advisory board of physicians who have extensive experience in managing patients with Gaucher disease, and who in part make up the authorship of this report.
Cerezyme, Genzyme's bioengineered human enzyme replacement therapy to treat Type 1 Gaucher disease, is currently used to treat more than 3,400 Gaucher disease patients in over 55 countries. Cerezyme is indicated for long-term enzyme replacement therapy for patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, and hepatomegaly or splenomegaly. Improvement and prevention of bone pain and crises are not included in the FDA-approved prescribing information.
Side effects 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 side effects include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Because Cerezyme therapy is administered by intravenous infusion, reactions at the site of injection may occur: discomfort, itching, burning, swelling or uninfected abscess. Symptoms suggestive of allergic reaction include anaphylactoid reaction (a serious allergic reaction), itching, flushing, hives, an accumulation of fluid under the skin, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. Approximately 15% of patients have developed immune reactions (antibodies); periodic monitoring by a physician is suggested. Patients should notify a physician immediately if they experience any side effects with treatment. For more information, consult a physician. To learn more, please see full product information, contact Genzyme at 1-800-745-4447, or visit www.cerezyme.com. Cerezyme is available by prescription only. |
