Velaglucerase alfa for Gaucher disease in Japan: Long-term post-marketing surveillance data from a unique population with high rates of neuronopathic subtypes
6 affiliations
- 1Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo, Japan
- 2Department of Pediatrics, Graduate School of Medicine, Juntendo University, Tokyo, Japan
- 3Diagnostics and Therapeutics of Intractable Diseases, Intractable Disease Research Center, Graduate School of Medicine, Juntendo University, Tokyo, Japan
- 4Department of Metabolism, Chiba Children's Hospital, Chiba City, Japan
- 5Trustee, The Jikei University School of Medicine, Tokyo, Japan
- 6Patient Safety & Pharmacovigilance Japan, Takeda Development Center Japan, Takeda Pharmaceutical Company Limited, Osaka, Japan
Abstract
Gaucher disease (GD) is a rare, autosomal recessive disease with reduced β-glucocerebrosidase activity, leading to symptoms including hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. Velaglucerase alfa is a native human β-glucocerebrosidase preparation approved as enzyme replacement therapy for Type 1 GD in many countries and for all GD types, including neuronopathic Type 2 and Type 3, in Japan. In this report, we provide the final (up to 8 years) safety and somatic effectiveness results of an all-case, open-label, post-marketing surveillance study in Japan. Among all patients, the incidence of adverse drug reactions (ADRs) was 23.3% (14/60 patients) and serious ADRs was 8.3% (5/60 patients). In the safety analysis population (excluding patients who did not provide consent for publication), ADR incidence was 19.5% (8/41 patients), most commonly vomiting and positive antibody test (4.9% [2/41 patients] each); one patient experienced vomiting as a serious ADR. ADRs were more common in Type 2 (7/14 patients) compared with Type 1 (1/12 patients) and Type 3 (0/15 patients). Three patients (7.3%), all Type 2, had five infusion-related reactions of pyrexia, vomiting (serious), swelling of eyelid, and hypersensitivity; all resolved or recovered. In the effectiveness analysis population (patients with any effectiveness data; n = 39), hemoglobin concentration, platelet count, and plasma angiotensin-converting enzyme concentration improved by Week 48 in patients with no prior GD treatment and were maintained in patients with prior GD treatment. These results support long-term use of velaglucerase alfa in Japanese patients with all GD types, with safety and somatic effectiveness consistent with previous findings.
Related
- Durability and Safety of PULSTA Transcatheter Pulmonary Valve: Insights From Five-Year Nationwide Post-Marketing Surveillance Study in Korea
- A Retrospective Post-Marketing Surveillance Study on TGlobulin25®, the First Rabbit Anti-Thymocyte Globulin (r-ATG) Biosimilar Worldwide, as Induction Therapy in Adult Kidney Transplant Recipients.
- Subtypes of invasive intracholecystic papillary-tubular neoplasms: a propensity score-matched Surveillance, Epidemiology, and End Results analysis.
- Early Recognition of Patients with Neuronopathic Forms of Mucopolysaccharidosis Type II in Clinical Practice
- Temporal trends and geographical inequalities in congenital anomaly mortality in China, 2008–2021: a population-based surveillance study
BibTeX
@article{hoshino2026velaglucerase,
title = {{Velaglucerase alfa for Gaucher disease in Japan: Long-term post-marketing surveillance data from a unique population with high rates of neuronopathic subtypes}},
author = {Miyako Hoshino and Kei Murayama and Hiroyuki Ida and Erika Asano and Hiromichi Otaka},
journal = {Molecular Genetics and Metabolism Reports},
year = {2026},
month = oct,
doi = {10.1016/j.ymgmr.2026.101373},
url = {https://doi.org/10.1016/j.ymgmr.2026.101373},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Velaglucerase alfa for Gaucher disease in Japan: Long-term post-marketing surveillance data from a unique population with high rates of neuronopathic subtypes
AU - Miyako Hoshino
AU - Kei Murayama
AU - Hiroyuki Ida
AU - Erika Asano
AU - Hiromichi Otaka
PY - 2026
DA - 2026/10/29
JO - Molecular Genetics and Metabolism Reports
DO - 10.1016/j.ymgmr.2026.101373
UR - https://doi.org/10.1016/j.ymgmr.2026.101373
AB - Gaucher disease (GD) is a rare, autosomal recessive disease with reduced β-glucocerebrosidase activity, leading to symptoms including hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. Velaglucerase alfa is a native human β-glucocerebrosidase preparation approved as enzyme replacement therapy for Type 1 GD in many countries and for all GD types, including neuronopathic Type 2 and Type 3, in Japan. In this report, we provide the final (up to 8 years) safety and somatic effectiveness results of an all-case, open-label, post-marketing surveillance study in Japan. Among all patients, the incidence of adverse drug reactions (ADRs) was 23.3% (14/60 patients) and serious ADRs was 8.3% (5/60 patients). In the safety analysis population (excluding patients who did not provide consent for publication), ADR incidence was 19.5% (8/41 patients), most commonly vomiting and positive antibody test (4.9% [2/41 patients] each); one patient experienced vomiting as a serious ADR. ADRs were more common in Type 2 (7/14 patients) compared with Type 1 (1/12 patients) and Type 3 (0/15 patients). Three patients (7.3%), all Type 2, had five infusion-related reactions of pyrexia, vomiting (serious), swelling of eyelid, and hypersensitivity; all resolved or recovered. In the effectiveness analysis population (patients with any effectiveness data; n = 39), hemoglobin concentration, platelet count, and plasma angiotensin-converting enzyme concentration improved by Week 48 in patients with no prior GD treatment and were maintained in patients with prior GD treatment. These results support long-term use of velaglucerase alfa in Japanese patients with all GD types, with safety and somatic effectiveness consistent with previous findings.
SN - 2214-4269
ER -