Vpriv (velaglucerase alfa) / Takeda - LARVOL DELTA

Phenotypic spectrum and treatment outcomes in Russian gaucher disease patients: Real-world experience with biosimilar imiglucerase (ASH 2025) - "Therapy: 321 patients received pathogenetic treatment: enzyme replacement therapy (ERT): 92% (imiglucerase-biosimilar [Russia]: 69%, velaglucerase: 30%, taliglucerase: 1%) substrate reduction therapy (eliglustat): 8% Outcomes: after 7 years of ERT, anemia persisted in 6% and severe thrombocytopenia (platelets 93% achieving hematologic stability on long-term therapy."

Clinical • Real-world • Real-world evidence • Gaucher Disease • Gene Therapies • Genetic Disorders • Hematological Disorders • Leukopenia • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Thrombocytopenia

Bone involvement in Gaucher disease: Data from a North African registry. (PubMed, Reumatol Clin (Engl Ed)) - "We presented descriptive data on BI derived from the Tunisian national Gaucher disease registry. This manifestation was common in our cohort. The limited size and heterogeneity of the treated subgroups precluded robust statistical comparisons. A major challenge in our setting is the delayed initiation of specific therapies, primarily due to late diagnosis and limited access to treatment."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Musculoskeletal Pain • Oncology • Osteoporosis • Pain • Rheumatology

Treatment of infants and very young children with Gaucher disease with velaglucerase alfa: a single-center experience (SSIEM 2023) - No abstract available

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders

A retrospective and prospective multicenter observational study of bone MRI changes in patients with type 1 Gaucher disease treated with velaglucerase alfa: the EIROS study. (SSIEM 2023) - No abstract available

Observational data • Retrospective data • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Treatment of infants and very young children with Gaucher disease with velaglucerase alfa: a single-center experience (SSIEM 2023) - No abstract available

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders

A retrospective and prospective multicenter observational study of bone MRI changes in patients with type 1 Gaucher disease treated with velaglucerase alfa: the EIROS study. (SSIEM 2023) - No abstract available

Observational data • Retrospective data • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Treatment of infants and very young children with Gaucher disease with velaglucerase alfa: a single-center experience (SSIEM 2023) - No abstract available

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders

A retrospective and prospective multicenter observational study of bone MRI changes in patients with type 1 Gaucher disease treated with velaglucerase alfa: the EIROS study. (SSIEM 2023) - No abstract available

Observational data • Retrospective data • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Treatment of infants and very young children with Gaucher disease with velaglucerase alfa: a single-center experience (SSIEM 2023) - P | "The data provide vital evidence on the effectiveness and safety of ERT with velaglucerase alfa in children 4 years old with GD and offer valuable insight into the presentation/course of GD in infants and very young children. (Study and medical writing funded by Takeda; intended for HCPs)"

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Pediatrics

A retrospective and prospective multicenter observational study of bone MRI changes in patients with type 1 Gaucher disease treated with velaglucerase alfa: the EIROS study. (SSIEM 2023) - P=N/A | "This study provided real-world evidence showing the long-term effectiveness of VELA for the treatment of GD, including for bone manifestations. The data suggest that if the assessment of MRI and BMB score by a radiologist with experience of GD bone manifestations is not possible, a simplified qualitative assessment provides sufficient evidence in daily clinical practice for the monitoring of bone disease progression and treatment response."

Observational data • Retrospective data • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Orthopedics • Thrombocytopenia • Type 1 Gaucher Disease

The French Gaucher disease registry: clinical features, complications, and treatment trends of 688 patients (SSIEM 2023) - "Among the currently treated patients (n=311), 57% received Imiglucerase, 18% Velaglucerase, and 25% Eliglustat. Parkinson`s disease developed in 10 (5%) patients at a median of 54 [46-62] years. Conclusion The registry enabled us to describe the epidemiology of GD in France, as well as the treatment evolution and the prevalence of GD complications and associated diseases."

Clinical • CNS Disorders • Fatigue • Gaucher Disease • Genetic Disorders • Hematological Malignancies • Hepatology • Lymphoma • Metabolic Disorders • Movement Disorders • Multiple Myeloma • Musculoskeletal Pain • Myeloproliferative Neoplasm • Oncology • Pain • Parkinson's Disease • Solid Tumor

Enzyme replacement therapy in infants and very young children with Gaucher disease using velaglucerase alfa: a single-center experience. (PubMed, Front Pediatr) - P | "No drug-related adverse events were recorded. These preliminary data suggest that velaglucerase alfa is well-tolerated and associated with improvements in clinical parameters in very young children with GD types 1 and 3, offering insights into the early presentation and course of GD in infancy and early childhood."

Journal • Gaucher Disease • Genetic Disorders • Hematological Disorders • Hepatology • Metabolic Disorders • Pediatrics • GBA

Real-World Observational Study of Velaglucerase Alfa (ChiCTR) - P=N/A | N=25 | Not yet recruiting | Sponsor: Beijing Childrens Hospital,Capital Medical University; Beijing Childrens Hospital,Capital Medical University

New trial • Real-world evidence • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Gaucher Disease Outcome Survey (GOS) (clinicaltrials.gov) - P=N/A | N=1257 | Recruiting | Sponsor: Shire | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders

EVALUATION OF ELIGLUSTAT THERAPY IN PATIENTS WITH GAUCHER DISEASE TYPE 1: A SINGLE CENTER EXPERIENCE (EHA 2025) - "More specifically, 3/12 had not received previous treatment (treatment-naïve, TN) while 9/12 had received enzyme treatment (treatment switch, TS); seven (7/9) had received imiglucerase and 2/9 velaglucerase. Our experience with the use of oral eliglustat in patients with GD1, despite the small number of patients, confirms that eliglustat treatment is an effective treatment for the disease."

Clinical • Anemia • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Thrombocytopenia • Type 1 Gaucher Disease

Bone involvement in Gaucher disease: Data from the tunisian registry (EULAR 2025) - "Thirty-one patients received substitutive enzymatic treatment with either velaglucerase alfa (n=22; 59%) or imiglucerase (n=9; 24%) as part of a clinical trial or compassionate use... Bone involvement was frequent in our cohort with poor response to treatment. This can be explained by the late start substitutive treatment in our patients due to diagnosis and access to therapies delay. In fact, most studies suggest that the greatest effect is seen in younger subjects during the period when peak bone densitometry is accrued."

Gaucher Disease • Genetic Disorders • Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Oncology • Orthopedics • Osteoporosis • Pain • Pediatrics • Rheumatology

WuXi Biologics Congratulates Partner CANbridge Pharmaceuticals on the Approval of Innovative Velaglucerase-beta for Injection (Gaurunning) for Gaucher Disease by China NMPA (PRNewswire) - "WuXi Biologics...congratulates its partner CANbridge Pharmaceuticals on the approval of its innovative velaglucerase-beta for injection (Gaurunning) by the China National Medical Products Administration (NMPA) for the treatment of adolescents aged 12 and above and adults with type I and type III Gaucher disease. This marks China's first and only locally developed enzyme replacement therapy (ERT) for Gaucher disease."

China approval • Gaucher Disease

Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified? (PubMed, Pharmacoecon Open) - "The study concludes that expanding ERT with either imiglucerase or velaglucerase to treat both Gaucher disease types 1 and 3b is not cost-effective at current prices in Thailand; however, it could become cost-effective with a reduction of approximately 60% in drug prices or if all eligible patients undergo HSCT."

Journal • Bone Marrow Transplantation • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Transplantation • Type 1 Gaucher Disease

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion (clinicaltrials.gov) - P=N/A | N=60 | Not yet recruiting | Sponsor: Takeda | Trial completion date: Sep 2026 ➔ Sep 2027 | Trial primary completion date: Sep 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys. (PubMed, Orphanet J Rare Dis) - P, P=N/A | "These findings suggest that self-administration of agalsidase alfa or velaglucerase alfa infusions are not associated with additional safety risks compared with HCP-supported infusions and are a suitable option for qualifying patients. Further research is warranted to support these findings and to explore further the long-term safety and efficacy of ERT self-administration. FOS trial registration: ClinicalTrials.gov, NCT03289065. Registered 01 April 2001, https://clinicaltrials.gov/study/NCT03289065 . GOS trial registration: ClinicalTrials.gov, NCT03291223. Registered 27 July 2010, https://classic."

Journal • Cardiovascular • Fabry Disease • Gastroenterology • Gastrointestinal Disorder • Gaucher Disease • Genetic Disorders • Infectious Disease • Metabolic Disorders

Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey. (PubMed, Orphanet J Rare Dis) - "In this study, changes in lyso-Gb1 concentrations from DBS were reflective of responses to enzyme replacement therapy initiation or withdrawal in most patients. These findings confirm that the use of DBS samples for routine monitoring of lyso-Gb1 concentrations in patients with GD is feasible in real-world settings and may be useful to assess treatment response."

Biomarker • Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Gaucher Disease Outcome Survey (GOS) (clinicaltrials.gov) - P=N/A | N=1257 | Recruiting | Sponsor: Shire | Trial completion date: Jan 2025 ➔ Sep 2025 | Trial primary completion date: Jan 2025 ➔ Sep 2025

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Effective encapsulation of therapeutic recombinant enzyme into polymeric nanoparticles as a potential vehicle for lysosomal disease treatment. (PubMed, Int J Biol Macromol) - "Finally, the viability of GD cell lines is not affected by NPs: Vela, in comparison with Velaglucerase alone, that negatively affects the viability of the target cells. This nanocarrier system for Velaglucerase alfa delivery in lysosomes, initially proposed to improve ERT for GD, may also serve as a starting point to address pathophysiological mechanisms in GD and other lysosomal disorders."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease (clinicaltrials.gov) - P3 | N=20 | Completed | Sponsor: Takeda | Active, not recruiting ➔ Completed

Trial completion • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry. (PubMed, J Clin Med) - P | "Most patients treated with enzyme replacement therapy or substrate reduction therapy reported improvements in clinical parameters within 1 year of treatment initiation, maintained over the course of treatment up to 12 years, whereas untreated patients had baseline values closer to standard reference thresholds and showed stability over time. The 12-year data from the GOS confirm the impact of long-term treatment with GD-specific agents and offer insights into disease progression and outcomes in a real-world setting."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases