A Phase 2 Multi-center, Open-label, Switch-over Trial to Evaluate the Safety and Efficacy of Abcertin® in Patients with Type 1 Gaucher Disease

  title={A Phase 2 Multi-center, Open-label, Switch-over Trial to Evaluate the Safety and Efficacy of Abcertin{\textregistered} in Patients with Type 1 Gaucher Disease},
  author={Jin-Ho Choi and Beom Hee Lee and Jung min Ko and Young Bae Sohn and Jin-Sung Lee and Gu-Hwan Kim and Sun-Hee Heo and June-Young Park and Yoo Mi Kim and Ja-Hye Kim and Han-Wook Yoo},
  journal={Journal of Korean Medical Science},
  pages={378 - 384}
Gaucher disease is a lysosomal storage disease for which enzyme replacement therapy has proven to be effective. A switch-over clinical trial was performed to evaluate the efficacy and safety of Abcertin® (ISU Abxis, Seoul, Korea) in subjects with type 1 Gaucher disease who were previously treated with imiglucerase. Five Korean patients with type 1 Gaucher disease were enrolled. Previous doses of imiglucerase ranged from 30 to 55 U/kg every other week. The same dose of Abcertin® was administered… 
A multicenter, open-label, phase III study of Abcertin in Gaucher disease
It is suggested that Abcertin can be an alternative ERT option for type 1 GD, and one patient developed anti-imiglucerase antibodies without neutralizing activity.
How we manage Gaucher Disease in the era of choices
The approach to the management of GD in the era of choices is presented, including a new algorithm for how to manage a newly diagnosed patient, with known efficacy and minimal toxicity.
Insight into Biosimilars: Short Description, Analytical Assessment and Market
The World Health Organization (WHO) defines a similar biotherapeutic product, i.e. a biosimilar as a biotherapeutic product that is similar in terms of quality, safety and efficacy to an already
Substrate reduction therapy as a new treatment option for patients with Gaucher disease type 1: A review of literatures
Gaucher disease type 1 (GD1) is an inherited lysosomal storage disorder caused by deficiency of acid β-glucosidase. The diminished enzyme activity...
The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease
This book aims to provide a chronology of key events and institutions that have contributed to the development of encephalopathy in children over a period of 40 years.
La maladie de Gaucher : quand y penser ?
Le diagnostic de maladie de Gaucher est confirme par la mise en evidence d’un deficit de l’activite de the glucocerebrosidase dans les leucocytes, ainsi que par l”identification des variants pathogenes des deux alleles du gene GBA1.
Recent advances and future challenges in Gaucher disease.


Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study
Velaglucerase alfa was generally well tolerated and effective for adults and children with GD1 in this study, and all disease‐specific parameters measured demonstrated clinically meaningful improvements after 12 months.
Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.
Enzyme replacement therapy is the standard of care for symptomatic Gaucher disease. Velaglucerase alfa is a human beta-glucocerebrosidase produced in a well-characterized human cell line. A 9-month
Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model
The responses of GC levels and storage cell numbers in Vela- and Imig-treated Gaucher mice at various doses provide a backdrop for clinical applications and decisions.
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
A position statement is presented based on the findings of the group about identification and monitoring of at-risk patients threatened by the abrupt withdrawal of treatment, the equitable distribution of residual imiglucerase - and access to alternative treatments including those that have completed phase III clinical trials but have not yet been licensed.
A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease.
GA-GCB demonstrated linear PK parameters over clinically relevant doses indicating that the dose of IV-administered GA- GCB to target tissues should also be linearly proportional to dose.
The long-term international safety experience of imiglucerase therapy for Gaucher disease.
Analysis of the long-term safety experience with imiglucerase therapy demonstrates a stable and low rate of adverse events and seroconversion from 1994 through 2005.
Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.
The results support safety and efficacy of taliglucerase alfa for Gaucher disease.
The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review.
The evidence suggests that the vast majority of the clinical characteristics of type I Gaucher's disease have little impact on subjective health-related quality of life (HRQoL) and that therefore for the majority of people with type I Goffman's disease this may not be a severe condition.
Replacement therapy with imiglucerase for type 1 Gaucher's disease
Low-dose low-frequency imiglucerase may be an alternative cost-effective approach with satisfactory clinical response and uncompromised quality of life in Gaucher's disease.
Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease.
Intravenous administration of macrophage-targeted glucocerebrosidase produces objective clinical improvement in patients with type 1 Gaucher's disease.