taliglucerase alfa

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (taliglucerase alfa) in Patients With Gaucher Disease

U.S. Food and Drug Administration Approves Protalix's Treatment Protocol for taliglucerase alfa
http://clinicaltrials.gov/ct2/show/NCT00962260?term=protalix&rank=3

Status: Available

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD), which have been mapped to chromosome 1 q21-q31, leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This accumulation leads to the visceral manifestations of hepatosplenomegaly, anemia and thrombocytopenia, as well as to the skeletal features and less frequently also to lung involvement.

Taliglucerase alfa is a plant cell expressed recombinant glucocerebrosidase enzyme for the treatment of Gaucher disease. Expression of proteins in plant cell culture is highly efficient, does not require post-expression modification of the protein, and is not susceptible to contamination by agents such as viruses that are pathological to humans.

taliglucerase alfa safety will be observed in this treatment protocol of patients with non-neuronopathic Gaucher disease who require enzyme replacement therapy. Eligible patients will receive intravenous (IV) infusions of taliglucerase alfa every two weeks. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage. The infusions will be administered at the selected medical center (information obtained from www.clinicaltrials.gov).

The following information was obtained from Protalix's website at www.protalix.com

taliglucerase alfa...
our lead proprietary product candidate, is a plant cell expressed recombinant Glucocerebrosidase enzyme (GCD) for the treatment of Gaucher disease. We have reached an agreement with the United States Food and Drug Administration on the final design of the pivotal phase iii clinical trial for taliglucerase alfa through the FDA's Special Protocol Assesment (SPA) process. We have completed enrollment for this study in December 2008 and expect to report results of the clinical trial in the second half of 2009.

We anticipate submitting a New Drug Application (NDA) for taliglucerase alfa to the FDA and other comparable regulatory agencies in other countries in the fourth quarter of 2009. We are treating patients in our pivotal phase III clinical trial in North America, South America, Israel, Europe and South Africa. In addition, we have started enrolling for a Switch Over study to assess the safety and efficacy of taliglucerase alfa. The trial is designed to include 15 patients with Gaucher disease that are currently undergoing enzyme replacement therapy with imiglucerase (Cerezyme®). patients with stable disease will be switched from intravenous imiglucerase (Cerezyme®) treatment every two weeks to intravenous infusions of taliglucerase alfa every two weeks for a nine-month period.

The taliglucerase alfa dose administered to each patient will be equal to the patient's previous imiglucerase dose and the infusions will be administered at selected investigational sites. The switchover-study is not a prerequisite for approval of taliglucerase alfa. At the end of the nine-month treatment period, all eligible patients will be offered the opportunity to enroll in the Company's on-going extension study. We expect to announce top line results of our pivotal phase three study during the second half of this year.