Elelyso (taliglucerase alfa) / Pfizer - 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

Economic Burden of Gaucher Disease: A Systematic Literature Review (ISPOR-EU 2025) - "In the US, the total annual direct costs per patient in 2021 ranged from $243,281 to $746,747, with treatment using taliglucerase alfa in adults having the highest financial impact... GD imposes a substantial economic burden worldwide, with significant variation in treatment costs across countries. Our findings emphasize the need for cost-effective management strategies and further research to reduce the financial impact on healthcare systems and patients globally."

HEOR • Review • Fatigue • Gaucher Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pediatrics • Rare Diseases

Ten-Year Follow-Up of Taliglucerase Alfa in Type 1 Gaucher Disease: Real-World Evidence from Albania. (PubMed, J Clin Med) - " Taliglucerase alfa offers sustained long-term clinical, hematological and biochemical benefits in both treatment-naïve and previously treated Gaucher disease type 1 patients, with a favorable safety profile. Glucosylsphingosine proved to be a highly sensitive biomarker for monitoring therapeutic efficacy and detecting treatment response."

HEOR • Journal • Real-world evidence • Gaucher Disease • Genetic Disorders • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease • GBA1

Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series. (PubMed, Mol Genet Metab Rep) - "Thus, 19 patients were switched from imiglucerase to taliglucerase, but more than a quarter experienced significant side effects. Here, we summarize these patients' clinical course and describe 6 suspected product-related adverse-effects."

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

Plant molecular farming: a promising frontier for orphan drug production. (PubMed, Biotechnol Lett) - "The FDA approval of plant-derived proteins for the treatment of Gaucher disease (Elelyso) and Fabry disease (Elfabrio) highlights the potential of plant-based expression systems for the development of suitable drugs targeting niche and orphan diseases. This review examines the potential of the plant system in producing orphan drugs and also highlights the opportunities and challenges related to orphan drug manufacturing."

Journal • Orphan drug • Review • Fabry Disease • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases

ORPHAN DRUGS IN ALZHEIMER'S DISEASE: CROSSROAD OF AMYLOID PATHOLOGY AND LYSOSOMAL STORAGE DISEASES (ADPD 2025) - "To test this hypothesis the effects of cerliponase alfa and taliglucerase alfa on A β accumulation in mouse hippocampal neurons (HT -22 neuronal cells) exposed to fAβ1-42 (a toxic fragment of full -length A β) and markers of autophagy -lysosome pathway related to AD were investigated. Therefore, we suggest that these orphan drugs could be considered promising novel therapeutics for neurodegenerative diseases in which autophagy pathways are impaired."

Alzheimer's Disease • CNS Disorders • Gaucher Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • ATG5 • GBA1 • mTOR

BRAZILIAN EXPERIENCE WITH TALIGLUCERASE ALFA FOR GAUCHER'S DISEASE: DESCRIPTIVE RESULTS (SSIEM 2024) - "This analysis suggests that TA therapy is a safe and effective treatment option for patients with type 1 GD, including for patients switched from other ERT."

Cough • Dermatology • Gaucher Disease • Immunology • Metabolic Disorders • Musculoskeletal Pain • Pain • Pediatrics • Rare Diseases • Respiratory Diseases • Urticaria

Taliglucerase alfa in the longterm treatment of children and adolescents with type 1 Gaucher disease: the Albanian experience. (PubMed, Front Pediatr) - "Out of the 1,301 total administrations, our patients reported only 37 (2.8%) infusion-related adverse events which were mild and transitory. Taliglucerase alfa exhibits good efficacy and a safe profile in the treatment of children and adolescents with Type 1 Gaucher disease."

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

Green Biologics: Harnessing the Power of Plants to Produce Pharmaceuticals. (PubMed, Int J Mol Sci) - "Plants have proved that they can produce life-saving therapeutic proteins (Elelyso™-Gaucher's disease treatment, ZMapp™-anti-Ebola monoclonal antibodies, seasonal flu vaccine, Covifenz™-SARS-CoV-2 virus-like particle vaccine); however, some of these therapeutic proteins are difficult to bring to market, which leads to serious difficulties for the manufacturing companies...The versatility of plant-derived biologics across diverse fields, such as human and animal health, industry, and agriculture, is emphasized. This review also meticulously examines regulatory considerations specific to plant-derived biologics, shedding light on the disparities faced compared to biologics produced in other systems."

Journal • Review • Ebola Virus Disease • Gaucher Disease • Infectious Disease • Metabolic Disorders • Novel Coronavirus Disease • Respiratory Diseases

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

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (clinicaltrials.gov) - P4 | N=14 | Completed | Sponsor: Ari Zimran | Active, not recruiting ➔ Completed

Trial completion • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Real-World Experiences with Taliglucerase Alfa Home Infusions for Patients with Gaucher Disease: A Global Cohort Study. (PubMed, J Clin Med) - "Twelve serious AEs in five separate case reports were considered treatment related: one case of chest discomfort/pain and hypertension and one case of erythema associated with a toe blister, for which causality could not be excluded; pain in extremity; projectile vomiting and chills, alongside excessive eye blinking; and an infusion-related AE (pruritus). In conclusion, this real-life global study demonstrated that taliglucerase alfa home infusions are safe with high compliance rates."

Journal • Real-world • Real-world evidence • Cardiovascular • Dermatology • Gaucher Disease • Genetic Disorders • Hypertension • Metabolic Disorders • Pain • Pruritus

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

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=40 | Not yet recruiting | Sponsor: AVROBIO

Gene therapy • New P2/3 trial • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders

The Bone Biomarker of Quantitative Chemical Shift Imaging in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long-Term Enzyme Replacement Therapy. (PubMed, J Clin Med) - "A minority of such patients with suboptimal bone response require therapeutic change. The next phase of the study will address the effect of switching to taliglucerase alfa on bone status for patients with less than optimal QCSI scores (<0.30)."

Biomarker • Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy (ASH 2018) - P=N/A; "In treatment-naïve patients, mean hemoglobin concentration and platelet counts increased. In conclusion, taliglucerase alfa was well tolerated for up to 33 months of treatment and demonstrated a durable therapeutic effect, as evidenced by stable or improved hemoglobin concentration and platelet counts in this expanded access study."

Clinical • Biosimilar • Type 1 Gaucher Disease

Budget Impact Analysis of Gaucher Disease Pharmacologic Treatment in Colombia (ISPOR-EU 2022) - "This study developed a Budget Impact Analysis of including Enzyme Replacement Therapy - ERT (Imiglucerase, Taliglucerase, Velaglucerase) and Substrate Reduction Therapy - SRT (Eliglustat) for type I Gaucher Disease (GDI) from the health system perspective over a 3-year time horizon in enrolled Colombian population. Therapy of choice and its frequency affect the most on budget impact in the health system. Almost all GDI cost burden is due to medications and treatment mix."

HEOR • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs (clinicaltrials.gov) - P=N/A | N=30 | Completed | Sponsor: Shaare Zedek Medical Center | Unknown status ➔ Completed

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

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (clinicaltrials.gov) - P4 | N=14 | Active, not recruiting | Sponsor: Ari Zimran | Recruiting ➔ Active, not recruiting | Trial primary completion date: Jun 2022 ➔ Jul 2023

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

A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease. (PubMed, Ann Pharmacother) - "The studies evaluated imiglucerase, velaglucerase alfa, taliglucerase alfa, miglustat, and eliglustat...Based on the assessed evidence, miglustat did not achieved expressive results. However, all evidence should be interpreted considering its limitations and does not replace well-conducted randomized trials."

Journal • Observational data • Review • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases

The budget impact of enzyme replacement therapy in type 1 Gaucher disease in the United States. (PubMed, J Med Econ) - "Across analyses, cost savings were realized with velaglucerase alfa compared with imiglucerase ($115,909) and taliglucerase alfa ($80,401)...The per-member per-month costs decreased by $0.0241 across years 1-3. BIM results show that increased velaglucerase alfa uptake for GD1 treatment is cost saving for US health plans."

HEOR • Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (clinicaltrials.gov) - P4 | N=15 | Recruiting | Sponsor: Ari Zimran | Trial completion date: Aug 2022 ➔ Aug 2023

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

Systematic Review and Meta-Analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease: Focus on Hematological Parameters (ISPOR 2022) - "Several meta-analyses were conducted for each drug: imiglucerase (6 months to 5 years), velaglucerase alfa (6 months to 7 years), taliglucerase alfa (6 months to 5 years), miglustat (6 months to 4 years), and eliglustat (6 months to 9 years). long-term results showed that ERTs are effective as treatment in both naïve and experienced patients. Miglustat did not significantly improve hemoglobin values in naïve patients and resulted in the decrease of platelet levels in experienced patients. All this evidence should be interpreted considering its limitations and does not replace a well conducted randomized trial."

Observational data • Review • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders

Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS). (PubMed, Orphanet J Rare Dis) - "The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa."

Journal • Real-world evidence • Gaucher Disease • Genetic Disorders • Metabolic Disorders

2022 DIC Wang award lecture: Plants as molecular foundries: From earth to deep space (ACS-Sp 2022) - "Although the first FDA-approved human therapeutic made in plant cell culture was introduced just ten years ago (ElelysoTM made in transgenic carrot cultures), plant cell culture offers a number of advantages: simple, chemically-defined, low cost animal component-free medium, the ability to produce complex glycoproteins, potential for long-term operation, and inability to propagate mammalian viruses. However, these systems are not without their own challenges. This presentation will describe some of the unique challenges encountered when using plants as molecular foundries, as well as our group’s research over the years to increase productivity, product quality, and production speed, with applications ranging from medical countermeasures for biodefense on Earth to molecular pharming on Mars."