Gaucher's Disease News and Research

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Gaucher disease is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain.

Protalix receives FDA CRL for taliglucerase alfa NDA for Gaucher disease

Protalix BioTherapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the Company's New Drug Application (NDA) for taliglucerase alfa for the treatment of Gaucher disease. Taliglucerase alfa is a plant-cell expressed form of glucocerebrosidase (GCD).

25 Feb 2011

Protalix: New data from taliglucerase alfa, oral GCD trials to be presented at Lysosomal Disease Network Symposium

Protalix BioTherapeutics, Inc., announced today that new clinical data from the switchover trial of taliglucerase alfa in Gaucher disease patients and preclinical data on oral enzyme glucocerebrosidase (oral GCD) will be presented today at the 7th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2011 in Las Vegas, Nevada.

19 Feb 2011

Genzyme reports 3-year follow-up data from eliglustat tartrate Phase 2 trial for Gaucher disease

Genzyme Corporation today announced three-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including bone disease, at the three-year timepoint.

19 Feb 2011

Genzyme's diagnostic products sold to Sekisui Chemical for $265 million

Genzyme Corporation today announced that it has completed the sale of its diagnostic products business to Sekisui Chemical Co., Ltd. for $265 million in cash.

1 Feb 2011

Genzyme to expand Geel facility to support growth of Myozyme and Lumizyme for Pompe disease

Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term growth of Myozyme® and Lumizyme® for Pompe disease.

20 Jan 2011

Positive results from Neuraltus NP002 Phase 1/2 study for levodopa-induced dyskinesias in Parkinson's disease

Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet medical needs, primarily for the treatment of neurodegenerative diseases, announced today top-line results from the Company's Phase 1/2 clinical study of NP002 for the treatment of dyskinesias (muscle movement disorders) resulting from levodopa therapy for patients with Parkinson's disease.

4 Dec 2010

Health Canada approves VPRIV enzyme replacement therapy for type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, announces that Health Canada has approved VPRIV (velaglucerase alfa), an enzyme replacement therapy (ERT) for long-term use in pediatric and adult type 1 Gaucher disease.

1 Dec 2010

Neuraltus reports positive results from NP001 Phase 1 study for ALS

Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet medical needs, primarily for the treatment of neurodegenerative diseases, announced today top-line results from the Company's Phase 1 clinical study of NP001 for the treatment of Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).

30 Nov 2010

Pfizer, Protalix announce marketing authorization submission in Europe for taliglucerase alfa

Pfizer Inc. and Protalix BioTherapeutics, Inc. today announced the submission of a Marketing Authorization Application to the European Medicines Agency for taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD)for the treatment of Gaucher disease. Taliglucerase alfa was granted Orphan Designation by the European Commission for the treatment of Gaucher disease on March 23, 2010.

29 Nov 2010

Positive preliminary data from Protalix's taliglucerase alfa switchover trial for Gaucher disease

Protalix BioTherapeutics, Inc., announced today positive preliminary data from the first 15 patients that completed the Company's nine month, worldwide, multi-center, open-label, switchover trial of taliglucerase alfa for the treatment of Gaucher disease under a protocol cleared by the U.S. Food and Drug Administration (FDA). The data indicate that patients can safely be switched to taliglucerase alfa from imiglucerase (Cerezyme®).

2 Nov 2010

Genzyme third quarter revenue increases to $1.0 billion

Genzyme Corp. today reported third-quarter earnings growth driven by increased shipments of Cerezyme® (imiglucerase for injection). Patients in the United States began returning to normal dosing levels last month, and patients globally are expected to be able to do so this quarter. Earnings growth in the third quarter was also driven by strong revenue from Lumizyme™ (alglucosidase alfa) and cost reduction measures.

21 Oct 2010

New disease pathways involving more than one cell type lead to Type 1 Gaucher disease: Study

Researchers at Yale School of Medicine have found that new disease pathways involving more than one cell type leads to Type 1 Gaucher disease, a rare genetic disorder in which fatty substances called glycosphingolipids accumulate in cells, resulting in liver/spleen enlargement, osteoporosis, bone pain, and increased risk of cancer and Parkinson's disease.

20 Oct 2010

Neuraltus achieves last patient out for NP001, NP002 studies

Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet needs, primarily in the treatment of neurodegenerative diseases, announced today the Company has achieved last patient out in two studies: a Phase 1 clinical study of NP001 in patients with Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease) and a Phase 1/2 clinical study of NP002 in patients with L-dopa-induced dyskinesias (muscle movement disorders).

5 Oct 2010

Genzyme responds to Sanofi-Aventis' unsolicited acquisition proposal

Genzyme Corp. today confirmed that it has received an unsolicited, non-binding proposal from Sanofi-Aventis to acquire all the outstanding shares of Genzyme for $69 per share in cash. The Genzyme board of directors met last night, unanimously affirmed its previous rejection of Sanofi's proposal, and instructed the company to send Sanofi the following response letter:

30 Aug 2010

Shire receives European marketing approval for VPRIV therapy to treat type 1 Gaucher disease

Shire, the global specialty biopharmaceutical company, announced today that the European Commission has granted marketing authorisation for VPRIV(R) (velaglucerase alfa), a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of type 1 Gaucher disease.

27 Aug 2010

Genzyme announces publication of phase 2 clinical trial results of eliglustat tartrate for Gaucher disease

Genzyme Corporation announced today that the two-year follow-up results from the phase 2 clinical trial of its investigational therapy known as eliglustat tartrate have been accepted for publication in the journal Blood. The results have been pre-published on the journal's website and are available to subscribers.

23 Aug 2010

Genzyme second-quarter revenue decreases to $1.08 billion

Genzyme Corp. today reported that second-quarter revenue was $1.08 billion, compared with $1.23 billion in the same period last year, reflecting limited shipments of Cerezyme® (imiglucerase for injection) and Fabrazyme® (agalsidase beta) due to product supply constraints in 2010. The company expects to be able to increase shipments of Cerezyme and Fabrazyme during the second half of the year.

21 Jul 2010

Protalix BioTherapeutics' taliglucerase alfa for Gaucher disease granted French ATU

Protalix BioTherapeutics, Inc. announced today that the French regulatory authority has granted an Autorisation Temporaire d'Utilisation (ATU), or Temporary Authorization for Use, for taliglucerase alfa for the treatment of Gaucher disease. An ATU is the regulatory mechanism used by the French Health Products and Safety Agency to make non-approved drugs available to patients in France when a genuine public health need exists.

13 Jul 2010

FDA accepts Protalix's taliglucerase alfa NDA, assigns PDUFA action date

Protalix BioTherapeutics, Inc., announced today that the Company's New Drug Application (NDA) for taliglucerase alfa has been accepted for review by the U.S. Food and Drug Administration (FDA). The FDA granted taliglucerase alfa a standard review time of ten months, assigning a Prescription Drug User Fee Act (PDUFA) action date of February 25, 2011.

12 Jul 2010

Shire presents positive data from TKT-025 EXT Phase I/II study for VPRIV in type 1 Gaucher disease patients

Shire plc, the global specialty biopharmaceutical company, today announced positive data from its long-term extension to its open label Phase I/II study (TKT-025 EXT) for VPRIV® (velaglucerase alfa), the company's enzyme replacement therapy for patients with type 1 Gaucher disease. The data were presented at the 9th Annual European Working Group on Gaucher Disease (EWGGD) in Cologne, Germany, and add to the growing body of clinical evidence which supports the safety and sustained efficacy of VPRIV.

2 Jul 2010