Genzyme Corporation, a biotechnology company, engages in the discovery and development of products and services primarily in the areas of rare genetic diseases, multiple sclerosis, cardiovascular diseases, and endocrinology. It offers Cerezyme to treat gaucher disease type I; Fabrazyme to treat fabry disease; Aldurazyme for mucopolysaccharidosis I; Myozyme and Lumizyme for pompe disease; Renagel that reduces phosphorous levels in chronic kidney disease (CKD) patients who are on dialysis; Renvela for use in dialysis patients and patients with earlier stage CKD; Hectorol, a vitamin D2 treatment for secondary hyperparathyroidism in CKD patients; Thyrogen to use in thyroid cancer follow-up; Leuk...
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Ablynx Enters into an Exclusive Research Collaboration with Genzyme
May 18 15
Ablynx announced that it has entered into an exclusive research collaboration with Genzyme to investigate Nanobodies® against a target that plays an important role in multiple sclerosis (MS) and specifically aligns with Genzyme's early-stage MS research programs involving neuroprotection and CNS repair. The neurodegeneration observed in MS is not directly targeted by existing treatments. Genzyme's research efforts aim to address this unmet need by targeting the underlying causes of MS disease progression and by developing treatments to protect neurons and promote repair of CNS damage. Under the terms of the agreement, Genzyme will have the right to perform in vitro and in vivo research with Ablynx's Nanobodies in MS-relevant models in return for an exclusivity fee. Upon completion of these studies, Genzyme will have the option to negotiate a license agreement. Ablynx has already generated potent Nanobodies against the specific target of interest and confirmed their activity in pre-clinical models.
Genzyme Corporation Announces Encouraging Data from Phase III Multiple Sclerosis Study
May 4 15
Genzyme Corporation has announced encouraging magnetic resonance imaging, or MRI, data from the Phase III pivotal studies with Lemtrada in patients with relapsing remitting multiple sclerosis, or RRMS. In relapsing remitting multiple sclerosis (RRMS) patients treated with Lemtrada in the Phase III pivotal studies, MRI effects observed in the two-year trials were maintained through two additional years in the extension study (years three and four). After the initial two courses of treatment in the pivotal studies, which were given at month zero and at month 12, approximately 70% of Lemtrada patients did not receive additional Lemtrada treatment during the following three years, through month 48. The Phase III trials of Lemtrada were randomized, two-year pivotal studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif) in patients with RRMS who had active disease and were either new to treatment (CARE-MS I) or who had an inadequate response to another therapy (CARE-MS II). Through year four, the adverse event profile of Lemtrada was consistent with that observed during the pivotal studies. The rate of brain atrophy, as measured by brain parenchymal fraction (BPF), decreased progressively over four years among Lemtrada patients in CARE-MS I. Among CARE-MS II Lemtrada patients, the rate of brain atrophy decreased progressively over three years and remained low in year four. In both studies, the median yearly brain volume loss was less than -0.20% in years three and four, which was lower than what was observed during the two-year pivotal studies. In CARE-MS I and II, treatment with Lemtrada significantly reduced the risk of developing new lesions compared to interferon beta-1a. In the extension study, most of the Lemtrada-treated patients from CARE-MS I and II were free of new lesions and MRI activity in years three and four (approximately 70%). Safety results from the second year of the extension study were previously reported. No new risks were identified. The most common side effects of Lemtrada are rash, headache, pyrexia, nasopharyngitis, nausea, urinary tract infection, fatigue, insomnia, upper respiratory tract infection, herpes viral infection, urticaria, pruritus, thyroid gland disorders, fungal infection, arthralgia, pain in extremity, back pain, diarrhea, sinusitis, oropharyngeal pain, paresthesia, dizziness, abdominal pain, flushing, and vomiting. Other serious side effects associated with Lemtrada include autoimmune thyroid disease, autoimmune cytopenias, infections and pneumonitis. A risk management program incorporating education and monitoring helps support early detection and management of these identified risks.
FDA Grants Fast Track Designation to Genzyme’s Investigational Substrate Reduction Therapy for the Treatment of Fabry Disease
Apr 28 15
Genzyme announced that the Food and Drug Administration (FDA) has granted Fast Track designation for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy for the treatment of Fabry disease. FDA’s Fast Track Drug Development Program is designed to facilitate frequent interactions with the FDA review team to expedite the clinical development and review of a New Drug Application (NDA) for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs for such disease or conditions. It also provides the opportunity to submit sections of an NDA on a rolling basis before a sponsor submits the complete application. Genzyme is currently enrolling patients in its Phase 2a trial of GZ/SAR402671, and plans to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter study. Fabry disease is a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance (called globotriaosylceramide, also referred to as GL-3 or Gb3) throughout the body. GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.