Data recently published in the Orphanet Journal of Rare Diseases
showed that long-term improvements and stability can be achieved with taliglucerase alfa in patients with Gaucher disease.
Taliglucerase alfa, commercially known as Eleyso, is developed by Protalix and Pfizer. It was approved by the U.S. Food and Drug Administration (FDA) in May 2012 for use in adults with Gaucher disease, and a supplemental new drug application (sNDA) for pediatric use was granted approval in September of that same year.
Gaucher disease is a genetic lysosomal storage disorder in which a deficiency of the glucocerebrosidase enzyme leads to a buildup of the lipid glucerebrosidase in the lysosomes. The accumulation become encorged Gaucher cells and can cause multisystemic damage in organs and tissues, especially the spleen, liver, bone, platelets, and hemoglobin.
The drug is an enzyme replacement therapy (ERT) produced in carrot cells and is the first recombinant therapeutic protein produced in a plant-cell expression system to be approved for use in humans.
The data published analyzes findings from key studies evaluating taliglucerase alfa, each of which assessed safety and spleen volume, liver volume, platelet count, hemoglobin concentration, and biomarkers as measures of efficacy.
Across 6 phase 3 clinical studies in treatment-naïve or treatment-switched adult and pediatric patients, treatment with taliglucerase alfa has been proven to result in clinically and statistically significant improvements in the major clinical features of Gaucher type 1, the most common form of the rare disease. Across the studies, long-term improvements were observed in major disease parameters and biomarkers in treatment-naïve adult patients at taliglucerase alfa dose levels of 30 U/kg and 60 U/kg.
Through all the studies, from baseline through conclusion, taliglucerase alfa was well tolerated and adverse events (AEs) were generally mild/moderate in severity and transience. Treatment with taliglucerase alfa resulted in improvements (treatment-naïve patients) or stability (patients switched from imiglucerase) in visceral, hematologic, and biomarker parameters.
Pediatric patients who were switched from imiglucerase maintained clinical stability in visceral and hematologic parameters during 33 months of treatment. Treatment-naïve pediatric patients who received up to 3 years of treatment with taliglucerase alfa, continuous improvements were observed in the same parameters in biomarkers. Growth inhibition and pubertal delay have been observed in children with GD, and exploratory analyses of growth and development in pediatric patients in the phase 3 taliglucerase alfa studies trended toward improvement in height and weight, progression of pubertal status, and absence of bone crises.
Adult patients who made the change demonstrated disease stability or improvements after a total of up to 3 years of treatment with taliglucerase alfa, while treatment-naïve adults achieved the primary endpoint of reduction in spleen volume after 9 months of treatment based on magnetic resonance imaging (MRI).
While the clinical studies in treatment-naïve patients with Gaucher disease were limited by low patient numbers, the comprehensive data set supports treatment with taliglucerase alfa in adult and pediatric patients who are naïve to ERT or who have previously been treated with imiglucerase.
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