Secondary endpoints also improved:
- Platelet levels increased from baseline by an absolute difference of 41 percent compared with placebo ( P<0.0001)
- Hemoglobin levels increased from baseline by an absolute difference of 1.2 g/dL compared with placebo ( P<0.0006)
- Liver volume decreased from baseline by an absolute difference of seven percent compared with placebo ( P<0.0072)
- A statistically significant improvement in total bone marrow burden was observed among patients in the eliglustat tartrate arm compared to placebo, and all other markers of bone disease showed trends towards improvement.
In an additional, pre-specified, efficacy analysis of the percent change in spleen volume from baseline, a mean change of minus six percent was observed in the eliglustat tartrate arm compared with minus three percent in the Cerezyme arm. This analysis also met the criteria for non-inferiority.With regard to secondary endpoints, after one year, nearly all patients receiving eliglustat tartrate met the stability criteria for the individual components of the composite endpoint:
- 94 percent of patients met spleen volume criteria
- 95 percent of patients met hemoglobin levels criteria
- 93 percent of patients met platelet levels criteria
- 96 percent of patients met liver volume criteria
About eliglustat tartrateEliglustat tartrate, a novel glucosylceramide analog given orally, was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease. The concept was initially developed by the late Norman Radin, MD, from the University of Michigan. In pre-clinical studies, the molecule, developed with James A. Shayman, MD, also from the University of Michigan, has shown high potency and specificity. Initiation of the Phase 2 and 3 studies of eliglustat tartrate in Gaucher disease followed an extensive pre-clinical research effort and a Phase 1 program. Cerezyme Important Safety Information 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 or hepatomegaly or splenomegaly (enlarged liver or spleen). Approximately 15% of patients have developed immune responses (antibodies). These patients have a higher risk of an allergic reaction (hypersensitivity). Use Cerezyme® (imiglucerase for injection) carefully if you have had an allergic reaction to the product in the past. Symptoms suggestive of allergic reaction happened in 6.6% of patients, and 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. 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, abdominal pain, vomiting, 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. Cerezyme is available by prescription only. For full prescribing information, please visit www.genzyme.com. About Genzyme, a Sanofi Company Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme’s portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com. About Sanofi Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and the new Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
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