Key Developments For Genzyme Corporation
Genzyme Corporation (GENZ.O) (Nasdaq)
Genzyme Corporation Announces Results Of Phase 2/3 Trial of Advanced Phosphate Binder
Genzyme Corporation announced results of a phase 2/3 study of its advanced phosphate binder (APB). The trial met its primary endpoint, which was to show that the APB lowered phosphate levels effectively compared to placebo. However, the APB did not show a significant improvement in phosphate lowering compared to the Company`s Renvela (sevelamer carbonate). Based on these results, the Company is not planning to pursue further clinical development of the APB. The Company was seeking to develop a product with higher potency that would more effectively bind phosphate, while maintaining all the benefits of Renvela. In the study, Renvela and the APB both effectively lowered patients` phosphorus levels. Most adverse events associated with both Renvela and the APB were mild in nature, and the most frequently reported events were gastrointestinal disorders. The trial was a randomized, double-blind, placebo-controlled study that enrolled 349 adult hemodialysis patients with hyperphosphatemia. It included seven arms: low, medium and high dose APB; low, medium and high dose Renvela, and placebo. It included a two-week screening period, a two-week washout period, and a three-week treatment period.
Genzyme Corporation Announces Data From Mipomersen Phase 3 Trial In hoFH Patients
Genzyme Corporation announced that data from the phase 3 study of mipomersen in patients with homozygous familial hypercholesterolemia (hoFH) were presented at the American Heart Association`s Scientific Sessions. The study met its primary endpoint in an intent-to-treat analysis, with a 25 percent reduction in LDL-cholesterol after 26 weeks of treatment, vs. 3 percent for placebo (p<0.001) which constitutes an average reduction greater than 100 mg/dL. The trial also met each of its secondary endpoints. The phase 3 study in hoFH patients, one of the trials to date in this rare population, was designed to test the efficacy and safety of adding mipomersen to substantial lipid-lowering therapy. Patients' average LDL-C at baseline was greater than 400 mg/dL. All but one of the patients were being treated with lipid-lowering therapy (50/51, 98%), of whom 11 (22%) were taking a statin alone and 39 (78%) were taking a statin in combination with at least one other lipid-lowering agent, most commonly ezetimibe (37/50, 74%). The LDL-C reductions observed in the study were in addition to those achieved with the patients` existing therapeutic regimen.
Genzyme Corporation Receives Complete Response Letter on Lumizyme Application
Genzyme Corporation announced that it has received a complete response letter from the FDA regarding its application to market Lumizyme (alglucosidase alfa) for the treatment of Pompe disease. In its letter, the agency stated that satisfactory resolution of deficiencies related to the Allston Landing manufacturing plant are required before the Lumizyme application can be approved. Genzyme believes the other elements of the Lumizyme review, such as the Risk Evaluation and Mitigation Strategy (REMS), the product label, and post-marketing requirements, have been satisfactorily addressed.
Genzyme Corporation Issues Letters to U.S. Healthcare Providers
Genzyme Corporation announced that it has issued letters to U.S. healthcare providers regarding its enzyme replacement products Cerezyme (imiglucerase for injection), Fabrazyme (agalsidase beta), Myozyme (alglucosidase alfa), Aldurazyme (laronidase), as well as Thyrogen (thyrotropin alfa for injection), filled at its Allston Landing manufacturing facility. The letters remind providers of the U.S. product labeling recommendations advising both visual inspection of vials and in-line filtration of enzyme replacement products to address the potential presence of foreign particles. The safety profile of these products remains unchanged. A review of the global safety database for these products has not identified any safety concerns to suggest that patients treated with Genzyme products have been exposed to foreign particles. However, a theoretical safety risk remains should a particle enter the bloodstream. Genzyme`s quality control processes have detected foreign particles in these products at a rate of approximately 1%. The Company`s rate has not increased over time. The global rate of product returns from customers due to the observance of foreign particles is less than approximately 0.02% for these products.
US FDA Says Some Genzyme Corporation's Drugs Contaminated-Reuters
Reuters reported that stainless steel fragments and other foreign particles were found in a small percentage of several of Genzyme Corporation rare disease drugs, U.S. regulators said. The particles were believed to be found in less than 1% of products based on product lots assessed to date, the Food and Drug Administration said in a statement. The FDA said the affected products included Cerezyme, which treats Gaucher disease. Other products were Fabrazyme for Fabry disease, Myozyme for Pompe disease, Aldurazyme for Mucopolysaccharidosis Type I, and Thyrogen, which is used in the diagnosis and treatment of thyroid cancer. No health problems linked to the contamination have been reported. The particles could, however, potentially cause serious adverse events in patients. These products are manufactured by a Genzyme's Allston Landing facility in Massachusetts, which is being investigated by the FDA.
