Researchers investigating the long-term efficacy and safety of taliglucerase alfa in treatment-naïve adults with Gaucher disease (GD) reported that the drug provided clinical benefits without new or severe side effects.
GD is a genetic disorder caused by a defective glucocerebrosidase enzyme that leads to a buildup of certain fats (lipids), specifically glucocerebroside, in the reticuloendothelial cells of the immune system. A standard therapy in managing GD, in use for over two decades, is based on enzyme replacement therapy (ERT) and aims to compensate for a patient’s lack of the enzyme.
Among currently available ERT drugs, a recombinant glucocerebrosidase developed by Protalix BioTherapeutics — called taliglucerase alfa (brand name, Elelyso) — is an approved ERT treatment for GD in a number of countries, including the United States, Israel, Brazil, Australia, Canada, and Chile.
The Phase 3 clinical trial evaluating taliglucerase (NCT00376168; Study PB-06-001) showed that GD patients receiving twice biweekly intravenous doses of 30 U/kg or 60 U/kg for nine months achieved a significant decrease in spleen and liver volumes, reduced chitotriosidase activity, and increased hemoglobin levels. Side effects were also reported to be mild to moderate.
Once that trial was finished, its patients were eligible to participate in an extension safety and efficacy study (NCT00705939; PB-06-003) at the same given dose of taliglucerase for an additional 15 months, after which time the drug’s use was unblinded and taliglucerase alfa treatment continued. A total of 23 patients completed the 36 months therapy in this additional extension study (NCT01422187; PB-06-007), receiving either dose of the drug every two weeks.
Results suggested that patients treated with taliglucerase alfa at 30 U/kg or 60 U/kg had reduced spleen volume from baseline of 50.1% and 64.6%, respectively, and reduced liver volume of 25.6% and 24.4%, respectively. Other results showed increased levels of hemoglobin by 16.0% and 35.8% (respective to a 30 U/kg or 60 U/kg dose), platelet count levels by 45.7% and 114.0%, respectively, and a decline in chitotriosidase activity by 71.5% and 82.2%, respectively. Finally, taliglucerase alfa was found to induce mild-to-moderate side effects, with the most common observed incidents being nasopharyngitis, headaches, pain in the extremities, arthralgia, hypertension, and upper respiratory tract infection.
“These long-term results of taliglucerase alfa demonstrated continued improvement in the clinical parameters with no new safety concerns through 36 months of treatment in treatment-naïve adult patients with GD. These results extend the clinical safety and efficacy profile of taliglucerase alfa in [these] patients,” the authors concluded.