Long-term safety and efficacy data of taliglucerase alfa, a Plant cell-expressed recombinant glucocerebrosidase, in the treatment of naïve Gaucher disease patients: 36-Month Results

Taliglucerase alfa is a beta-glucocerebrosidase enzyme replacement therapy (ERT) that is approved in the USA, Israel, and Uruguay for the treatment of Gaucher disease (GD) in adults, and is the first approved plant cell–expressed biotherapeutic. Pivotal study PB-06-001 was a 9-month, randomized, doubleblind, dose-ranging (30 and 60 U/kg) trial to assess the safety and efficacy of taliglucerase alfa in treatment-naive GD patients. Primary outcome measure was change from baseline in spleen volume, while secondary outcome measures included change from baseline in liver volumes, platelet counts, hemoglobin levels, and chitotriosidase activity. Key inclusion criteria were splenomegaly >8 multiples of normal (MN) and platelet counts <120,000/mm 3 . At the completion of the 9-month study, patients were eligible to enter an open-label extension study, PB-06-003, with taliglucerase alfa given every 2 weeks at the same dose as in study PB-06-001. Twenty-six treatment-naive GD patients from study PB06-001 continued double-blind treatment in study PB06-003. This interim analysis presents results for 23 patients that completed 27 months in PB-06-003 for a total of 36 months of taliglucerase alfa treatment. Mean spleen volume was reduced from 16.4/17.8 MN to 8.1/5.6 MN, liver volume was reduced from 1.7/1.5 to 1.3/1.1 MN, and chitotriosidase activity was reduced by 73.5% and 83.0% for 30 U/kg and 60 U/kg, respectively. Hemoglobin concentration and platelet counts were also improved. All treatment-related adverse events were mild or moderate in severity and transient in nature.