Vpriv (velaglucerase alfa) / Takeda - LARVOL DELTA

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

Gaucher Disease Outcome Survey (GOS) (clinicaltrials.gov) - P=N/A | N=1257 | Recruiting | Sponsor: Shire | Trial completion date: May 2024 ➔ Jan 2025 | Trial primary completion date: May 2024 ➔ Jan 2025

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

Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry. (PubMed, J Clin Med) - "There were 21 deaths overall, none of which were considered related to treatment. This analysis of data from the GOS registry supports the safety and efficacy of velaglucerase alfa in patients with GD."

Journal • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Thrombocytopenia

A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study. (PubMed, J Clin Med) - " The qualitative real-world data support findings from clinical trials suggesting the long-term effectiveness of velaglucerase alfa on GD1 bone manifestations. When MRI assessment by radiologists with experience of GD is not possible, a simplified qualitative assessment may be sufficient in clinical practice for monitoring bone disease progression and treatment response."

Journal • Real-world • Real-world evidence • Gaucher Disease • Genetic Disorders • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Orthopedics • Rare Diseases • Thrombocytopenia • Type 1 Gaucher Disease

Survey Study for Velaglucerase Alfa (VPRIV) in Japan (clinicaltrials.gov) - P=N/A | N=77 | Completed | Sponsor: Takeda | Trial completion date: Jan 2024 ➔ May 2024 | Trial primary completion date: Jan 2024 ➔ May 2024

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

Survey Study for Velaglucerase Alfa (VPRIV) in Japan (clinicaltrials.gov) - P=N/A | N=77 | Completed | Sponsor: Takeda | Active, not recruiting ➔ Completed

Trial completion • Gaucher Disease • Genetic Disorders • Metabolic Disorders

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 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

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

PEDS: A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease (clinicaltrials.gov) - P=N/A | N=11 | Completed | Sponsor: Takeda | Recruiting ➔ Completed | N=20 ➔ 11 | Trial completion date: Jan 2025 ➔ Apr 2023 | Trial primary completion date: Jan 2025 ➔ Apr 2023

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

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

Enrollment closed • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Skeletal Manifestations, Bone Pain, and BMD Changes in Albanian Type 1 Gaucher Patients Treated with Taliglucerase Alfa. (PubMed, J Osteoporos) - "Bone pain has gradually improved in all patients. Also, BMD values have been enhanced over six years of treatment, especially in children."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Musculoskeletal Pain • Pain • Type 1 Gaucher Disease

Gaucher Disease Outcome Survey (GOS) (clinicaltrials.gov) - P=N/A | N=1257 | Recruiting | Sponsor: Shire | Trial completion date: Dec 2023 ➔ May 2024 | Trial primary completion date: Dec 2023 ➔ May 2024

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

Survey Study for Velaglucerase Alfa (VPRIV) in Japan (clinicaltrials.gov) - P=N/A | N=77 | Active, not recruiting | Sponsor: Takeda | Completed ➔ Active, not recruiting | Trial completion date: Sep 2022 ➔ Jan 2024 | Trial primary completion date: Sep 2022 ➔ Jan 2024

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

Treatment of infants and very young children with Gaucher disease with velaglucerase alfa: a single-center experience (SSIEM 2023) - P=N/A | "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 • Hematological 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

An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 (clinicaltrials.gov) - P2/3 | N=0 | Withdrawn | Sponsor: AVROBIO | N=40 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Gene therapy • Trial withdrawal • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders

Rapid home therapy infusion of velaglucerase alfa in naïve patients with Gaucher disease. (PubMed, Intern Med J) - "Home therapy with rapid infusion of high-dose velaglucerase alfa was a safe, effective and preferable alternative for patients with GD naïve to treatment. We believe that shortening the infusion time improves the QoL of patients with GD who have a lifelong commitment to intravenous therapy."

Journal • Gaucher Disease • Genetic Disorders • Infectious Disease • Metabolic Disorders • Novel Coronavirus 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