Date: 2014-06-27
Type of information: Presentation of results at a congress
phase:
Announcement: presentation of results at the European Working Group on Gaucher Disease 2014 11th Meeting being held June 25-28 in Haifa, Israel
Company: Protalix BioTherapeutics (Israel)
Product: Elelyso® (taliglucerase alfa)
Action
mechanism: enzyme replacement therapy. Elelyso® is the first FDA-approved plant cell-based enzyme replacement therapy for Gaucher disease.
Disease: Gaucher disease
Therapeutic area: Rare diseases
Country:
Trial details:
Latest
news: * On June 27, 2014, Protalix BioTherapeutics announced that new clinical data on Elelyso® (taliglucerase alfa) will be presented at the European Working Group on Gaucher Disease 2014 11th Meeting being held June 25-28 in Haifa, Israel. Dr. Zimran is delivering an oral presentation titled "Taliglucerase alfa in adult patients with Gaucher disease who were previously treated with imiglucerase: 36-month safety and efficacy results". This presentation will describe long-term safety and efficacy data from the Company's multi-center, open-label switchover extension trial of Elelyso®. The Company's original switchover trial was a nine-month trial in which patients with stable disease were switched from treatment via intravenous infusions of imiglucerase (Cerezyme®) to intravenous infusions of Elelyso® every two weeks at an equivalent dose to the patient's previous imiglucerase dose. Patients who participated in the switchover trial were given the option to continue treatment with Elelyso® in the Company's switchover extension trial. A total of 14 patients completed the 36 months of treatments with taliglucerase alfa, at which point all patients remained clinically stable. The disease parameters that were evaluated were similar to baseline after 36 months of treatment, suggesting ongoing disease stability. These parameters included levels of hemoglobin: (Base line: 13.4 g/dL- 36 Months: 13.3 g/dL), platelet count: (Base line: 167,940 /mm3- 36 Months: 170,286 /mm3), spleen volume (Base line: 4.6 MN- 36 Months: 3.7 MN), liver volume (Base line: 1.0 MN- 36 Months: 1.0 MN), [MN = Multiples of the Normal size according to body weight] and chitotriosidase activity (reduction of -51.5% after 36 Months). In addition, adverse events were evaluated, all treatment-related adverse effects were mild or moderate in severity and transient in nature. The long-term safety and efficacy results demonstrate that Elelyso® has a well-established safety profile and is an effective alternative long-term treatment for adult Gaucher patients treated previously with imiglucerase. In addition, the full data from the initial nine-month multi-center, open-label switchover trial of ELELYSO for the treatment of Gaucher disease were just made available in the 18 June 2014 online publication of Blood Cells, Molecules, and Diseases in an article entitled "A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase". The full article can be found on the journal's website at http://www.journals.elsevier.com/blood-cells-molecules-and-diseases. * On February 13, 2013, Protalix BioTherapeutics has announced that new clinical data on Elelyso®(taliglucerase alfa) will be presented at the 9th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2013 being held February 13-15 in Orlando, Florida. Gregory Pastores, M.D., Professor of Neurology and Pediatrics and Director of the Neurogenetics Laboratory at the New York University School of Medicine, is presenting long-term data from the Company\'s multi-center, open-label switchover extension trial of Elelyso® for the treatment of Gaucher disease. The Company\'s original switchover trial was a nine-month trial in which patients with stable disease were switched from treatment via intravenous infusions of imiglucerase (Cerezyme®) to intravenous infusions of Elelyso® every two weeks at an equivalent dose to the patient\'s previous imiglucerase dose. Patients who participated in the switchover trial were given the option to continue treatment with Elelyso® in the Company\'s switchover extension trial. Twenty-five adult patients completed the switchover trial, of which 19 elected to continue treatment with Elelyso® through the long-term extension trial. Five of the six patients who did not enroll in the extension trial continued nonetheless to receive Elelyso® through the Company\'s various compassionate use programs. One patient was unable to comply with the study protocol and therefore was not eligible to participate in the extension trial. A 24 month interim analysis of the switchover trial demonstrates that all patients remained stable with regard to all key disease parameters, spleen volume, liver volume, platelet count and hemoglobin concentration, as well as the chitotriosidase activity biomarker after switching to Elelyso® from imiglucerase. The safety analysis presented for the 24-month switchover treatment duration demonstrates that Elelyso® was well tolerated, and no drug related serious adverse events were reported. One patient developed neutralizing IgG antibodies that were determined to be positive in an in vitro assay, and were determined to be negative in a cell-based assay. Four of the 19 patients enrolled in the extension trial discontinued treatment; one switched to the Elelyso® compassionate use program, one enrolled in another clinical trial, one was unable to comply with the study protocol and one was not pleased with that individual\'s personal results. In conclusion, the data demonstrates that Elelyso® has a well-established safety profile and is an effective alternative treatment for adult Gaucher patients treated previously with Cerezyme®.
