Plant cell-expressed recombinant glucocerebrosidase: Taliglucerase alfa as therapy for Gaucher disease in adults patients previously treated with imiglucerase: 24-month results

Abstract

Taliglucerase alfa is an enzyme replacement therapy (ERT) approved in the USA, Israel, and Uruguay for adult patients with type 1 Gaucher disease (GD). Study PB-06-002 was a 9-month, phase 3, multicenter, open-label, switchover trial that evaluated safety and efficacy of taliglucerase alfa in patients with GD previously treated with imiglucerase for at least 2 years. Eligible patients entered a 12-week evaluation period to establish the stability of their disease based on hematologic parameters. For patients whose imiglucerase regimen was changed due to drug shortage, eligibility was based on historical data of disease stability. Patients with stable disease were then switched from imiglucerase to the same dose of taliglucerase alfa. Efficacy was determined by evaluation of platelet counts, hemoglobin levels, and spleen and liver volumes for clinical deterioration. The control for this study was each patient’s previous historical clinical and stability laboratory measurements. At completion of the 9-month study, patients were eligible to enter an open-label extension study, PB06-003, with taliglucerase alfa given every 2 weeks at the same dose as in study PB-06-002. This is an interim report for the 15 adult patients who have completed 24 total months of treatment. Following 24 months’ treatment with taliglucerase alfa, efficacy parameters of spleen volume, liver volume, platelet counts, hemoglobin concentration, and chitotriosidase activity were maintained or improved in these patients following switchover to taliglucerase alfa. None of the patients met the criteria for clinically relevant deteriorations as specified by the study protocol. All treatment-related adverse events were mild or moderate in severity and transient in nature.