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Blood Cells Mol Dis. 2016 Jul 18. pii: S1079-9796(16)30087-0. doi: 10.1016/j.bcmd.2016.07.002. [Epub ahead of print]

Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naïve patients with Gaucher disease.

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Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel. Electronic address:
Pontificia Universidad Catolica de Chile, Santiago, Chile.
CIBERER, Hospital Universitario Miguel Servet, IIS Aragón, Zaragoza, Spain.
Rambam Medical Center, Haifa, Israel.
Sapienza University, Rome, Italy.
Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School, Belgrade, Serbia.
Centro Médico Nacional Siglo XXI, Servicio de Hematologia, Mexico City, Mexico.
Hospital de Especialidades No1, Leon, Mexico.
Faculty of Health Sciences, University of the Witwatersrand, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
Protalix BioTherapeutics, Carmiel, Israel.


Taliglucerase alfa, the first available plant cell-expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60U/kg every other week through 9months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30U/kg, n=8; 60U/kg, n=9; dose adjusted, n=2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (-8.7, -6.9, -12.4 multiples of normal), liver volume (-0.6, -0.4, -0.5 multiples of normal), chitotriosidase activity (-83.1%, -93.4%, -87.9%), and chemokine (CC motif) ligand 18 concentration (-66.7%, -83.3%, -78.9%), as well as mean increases in hemoglobin concentration (+2.1, +2.1, +1.8mg/dL) and platelet count (+31,871, +106,800, +34,000/mm3). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at as NCT01422187.


Anemia; Chemokine (CC motif) ligand 18; Chitotriosidase; Enzyme replacement therapy; Gaucher disease; Hepatomegaly; Splenomegaly; Taliglucerase alfa; Thrombocytopenia

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