Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy

Abstract

IntroductionGaucher disease is the first lysosomal disease to benefit from enzyme replacement therapy, thus serving as model for numerous other lysosomal diseases. Alglucerase was the first glucocerebrosidase purified from placental extracts, and this was then replaced by imiglucerase – a Chinese hamster ovary cell-derived glucocerebrosidase.AimThe aim was to review the evidence underlying the use of imiglucerase in Gaucher disease type 1Evidence reviewData from clinical trials and Gaucher Registries were analyzed.ConclusionImiglucerase has been prescribed and found to have an excellent efficacy and safety profile. We report herein the evidence-based data published for 26 years justifying the use of imiglucerase.

Retrospective Analysis of Whole-Body Magnetic Resonance Imaging of Bone Manifestations in Long-Term Treated Patients with Gaucher Disease Type 1

“…Though both groups benefitted from therapy, early start of treatment seems warranted, as irreversible complications were only visible in the "late" treatment group. This finding was consistent with current knowledge of Gaucher disease and consecutive treatment recommendations [1,10,23].…”

“…Several studies have demonstrated the effectiveness of ERT during the first years of treatment, based on MRI analyses [7,18]. Usually, after ERT initiation in Gaucher disease major response occurs within the first years [1,23], following a first order kinetic, while later on only small changes occur. Despite the relatively short follow-up after long-term ERT, our data also suggests at least disease stabilization with hints for possible slight improvements concerning bone involvement.…”

Gaucher disease: Progress and ongoing challenges

“…Striking and rapid reversal of many of the overt aspects of type 1 Gaucher disease were seen in pivotal clinical trials of alglucerase and and later with the recombinant form of the enzyme, imiglucerase (Genzyme, Cambridge, MA, USA) and have been replicated again and again in small series of patients from single centers as well as a global, industry-sponsored multinational registry comprising almost 6,000 patients that collectively harbor ~54,000 patient-years of enzyme therapy experience [106]. Enzyme replacement therapy has proven highly effective in reversing hepatosplenomegaly, cytopenia, osteopenia, especially in children and young adults, and in reducing the risk of avascular osteonecrosis [107].…”

Results of a phase I open randomized comparative crossover clinical trial to assess the safety and pharmacokinetics of Glurazim® (imiglucerase) in comparison with the reference product in healthy volunteers

“…имиглюцеразы сочетается с хорошей переносимостью и отсутствием выраженных побочных эффектов в лечении пациентов различного возраста с БГ 1-го и 3-го типов [7,9,10]. В июне 2009 г. фармацевтическая компания Джензайм Лтд.…”

“…Для 1-й и 2-й групп добровольцев статистически значимых различий между препаратами по показателям T 1 / 2 и K el не выявлено (p >0,05). Полученные в исследовании результаты по указанным фармакокинетическим параметрам согласуются с данными литературы по препарату имиглюцераза [9,10]. В настоящем исследовании не выявлено статистически значимого (p >0,05) влияния последовательности введения ИП -отсутствует эффект периода введения препарата, а также не отмечено парного взаимодействия период × препарат (см.…”