Extimia (empegfilgrastim biosimilar) / Biocad - LARVOL DELTA

Arvenacogene sanparvovec: A novel gene therapy for hemophilia B – results from A phase I–II first-in-human trial (ASH 2025) - P1/2 | "A single administration of ANB-002 (arvenacogene sanparvovec) gene therapy to 13 patients withmoderately severe or severe hemophilia B resulted in durable factor IX expression, sustained clinicalbenefit that made it possible completely discontinue the FIX prophylaxis for 10 from 13 participants.Therapy was well tolerated."

First-in-human • Gene therapy • P1/2 data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Network Meta-Analysis of Survival Outcomes in the First-Line Treatment of Unresectable or Metastatic Melanoma: Scoping Review of Methodology (ISPOR-EU 2025) - "NMA is widely used to compare treatments for unresectable or metastatic melanoma, but most studies lack modern methodological approaches, including unclear model selection, absence of proportional hazards testing, and reliance on aggregated data, which limits the accurate modelling of time-dependent effects and interpretability of results. Future research should adopt IPD NMA with flexible survival models for more reliable results."

Metastases • Retrospective data • Review • Melanoma • Solid Tumor

The Disease Burden of Systemic Sclerosis in the Russian Federation (ISPOR-EU 2025) - "Lack of pathogenetic therapies for SSc has forced patients to rely on largely ineffective treatments (nearly all drugs for the primary disease are used off-label). This contributes to the development of numerous life-threatening complications, that consume most direct medical costs and ultimately result in early death. Simultaneously, patients experience significant declines in work productivity, leading to unemployment and substantial economic losses."

Gene Therapies • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases • Scleroderma • Systemic Sclerosis

Predicting Outcomes and Estimating Minimum Sample Sizes for Divozilimab Efficacy in NMOSD: A Clinical Trial Simulation and Unanchored Matching-Adjusted Indirect Comparison (ISPOR-EU 2025) - "Simulation modelling predicts the 6-month ARR of 0.116 (90% CI: 0.030; 0.212) for Divozilimab, corresponding to ARR of 0.117 (90% CI: 0.050; 0.315) versus placebo. A minimum sample size of 35 patients receiving Divozilimab is predicted to provide ≥90% power to demonstrate superiority based on the primary objective."

Biomarker • Clinical • CNS Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • IL2RG

Adjusting to Nonoverlapping Variables in the Unanchored Matching-Adjusted Indirect Comparisons of Survival Outcomes: Application of Monte Carlo Simulations (ISPOR-EU 2025) - P3, P3b/4 | "OBJECTIVES: This research aims to compare overall (OS) and progression-free (PFS) survival between two anti-PD1+anti-CTLA4 combinations (prologolimab+nurulimab and nivolumab+ipilimumab) in the first line therapy of patients with advanced melanoma. Systematic literature review revealed three randomized clinical trials: OCTAVA (NCT05732805, 135 patients on prolgolimab+nurulimab (PROLGO+NURU)), CheckMate-067 (NCT01844505, 314 patients on nivolumab 1 mg/kg + ipilimumab 3 mg/kg (NIVO1+IPI3)), CheckMate-511 (NCT02714218, 178 patients on NIVO1+IPI3, 180 patients on nivolumab 3 mg/kg + ipilimumab 1 mg/kg (NIVO3+IPI1))... Monte-Carlo simulations are applicable for adjustment to non-overlapping variables in MAICs, although they require additional assumptions about survival distribution on the study treatment in unstudied populations, which are subject to further validation."

Melanoma • Mucosal Melanoma • Solid Tumor

Efficacy and Safety of Biologics to Treat Adults With Active Radiographic Axial Spondyloarthritis: Systematic Review and Bayesian Network Meta-Analysis (ISPOR-EU 2025) - "OBJECTIVES: To compare efficacy and safety of IL-17 inhibitors in the treatment of adults with active radiographic axial spondyloarthritis (r-AxSpA) over a 16-week period using network meta-analysis (NMA). We conducted systematic literature search in PubMed and Embase in November 2024 to retrieve publications with results of randomized controlled trials (RCTs) evaluating efficacy and safety of IL-17 inhibitors (netakimab, secukinumab, ixekizumab, bimekizumab, brodalumab, xeligekimab, vunakizumab) in adults with active r-AxSpA... Netakimab showed the highest relative efficacy among available IL-17 inhibitors used to treat adults with active r-AxSpA over 16-week horizon and demonstrated a favorable safety profile."

Retrospective data • Review • Ankylosing Spondylitis • Immunology • Inflammatory Arthritis • Seronegative Spondyloarthropathies • Spondylarthritis • IL17A

HEMATOPOIETIC STEM CELL MOBILIZATION USING PEG-G-CSF EMPEGFILGRASTIM (EXTIMIA®) IN PATIENTS WITH LYMPHOPROLIFERATIVE DISEASES: EXPERIENCE OF SEVERAL CENTERS IN THE RUSSIAN FEDERATION (EBMT 2025) - "The results of the first multicenter experience with the use of Extimia® BIOCAD (INN: empegfilgrastim) for the mobilization of autologous blood stem cells in patients with lymphoproliferative diseases demonstrate its high efficacy with a favorable safety and tolerability profile: 74% (66/89) of successful mobilizations after a single administration of a fixed dose of the drug."

Clinical • B Cell Lymphoma • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Multiple Myeloma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Plasmacytoma • Solid Tumor • T Cell Non-Hodgkin Lymphoma

DEFENDOR: Real-world evidence of primary prolonged G-CSF prophylaxis by empegfilgrastim for relative dose intensity compliance in patients with solid tumors—The final analysis. (ASCO 2024) - "Here we present the final results of a multicenter prospective observational post-marketing study of safety and efficacy of pegylated G-CSF Extimia (INN: empegfilgrastim, JSC BIOCAD) in pts with ST who receive cytotoxic therapy... Primary prolonged G-CSF prophylaxis by empegfilgrastim allows effectively maintaining RDI across various tumor types (TT) and treatment groups in pts with ST in routine clinical practice. Clinical trial information: NCT0481144."

Clinical • Compliance • HEOR • Real-world • Real-world evidence • Anemia • Chemotherapy-Induced Neutropenia • Hematological Disorders • Neutropenia • Oncology • Solid Tumor

Defendor: Real-world evidence of primary prolonged G-CSF prophylaxis by empegfilgrastim for relative dose intensity compliance in patients with solid tumors—The primary analysis. (ASCO 2022) - P | "This multicenter prospective observational post-registration study of prolonged G-CSF empegfilgrastim (Extimia) was designed to evaluate the RDI of the cytotoxic therapy course under PP by empegfilgrastim in patients (pts) with ST receiving myelosuppressive therapy in the routine clinical practice... Thus, PP with prolonged G-CSF empefilgrastim allows effectively maintain RDI in different nosology and treatment groups in pts with ST in the routine clinical practice."

Clinical • HEOR • Real-world evidence • Back Pain • Chemotherapy-Induced Neutropenia • Hematological Disorders • Infectious Disease • Musculoskeletal Pain • Neutropenia • Novel Coronavirus Disease • Oncology • Pain • Solid Tumor • CSF3

Incidence of severe and febrile neutropenia in cancer patients treated with myelosuppressive chemotherapy in real clinical oncology practice: Preliminary results from the FLAME study. (ASCO 2022) - "Primary prophylaxis was given only to 44.1% pts with a high risk of FN [empegfilgrastim (Extimia®) 19,1%, filgrastim 25%]... Preliminary results from this study suggest that the risk of FN is higher from the start of CT. Delaying and reducing the dose of CT is still typical for oncologists. This practice is associated with low adherence to guidelines."

Clinical • Febrile Neutropenia • Gastrointestinal Cancer • Gastrointestinal Disorder • Gynecologic Cancers • Hematological Disorders • Infectious Disease • Lung Cancer • Neutropenia • Oncology • Sarcoma • Solid Tumor

Def_Special: Defendor Special: A Multicenter Prospective Observational Post-registration Study of Combined Chemotherapy With Empegfilrastim Support to Evaluate Safety and Efficacy in Patients With High and "Gray Zone" Risk Reccurrence Breast Cancer, Gastointestinal Cancers and Gynecological Malignancies (clinicaltrials.gov) - P; N=285; Recruiting; Sponsor: Moscow Clinical Scientific Center

Clinical • New trial • Breast Cancer • Gastrointestinal Cancer • Gynecologic Cancers • Neutropenia • Oncology • Solid Tumor • HER-2

DEFENDOR: A Multicenter Prospective Observational Post-registration stuDy of Extimia® (INN: empEgfilrastim) to Evaluate Efficacy and saFEty in patieNts With soliD tumORs (clinicaltrials.gov) - P; N=500; Recruiting; Sponsor: Biocad

Clinical • New trial • Neutropenia • Oncology • Solid Tumor

Efficacy and tolerability of granulocyte colony-stimulating factors in cancer patients after chemotherapy: A systematic review and Bayesian network meta-analysis. (PubMed, Sci Rep) - "S-G-CSF biosimilar, empegfilgrastim, and long-acting G-CSF (L-G-CSF) biosimilar were best G-CSF drugs in reducing severe neutropenia (SN) (cumulative probabilities: 21%, 20%, 15%, respectively). Mecapegfilgrastim, balugrastim, lipegfilgrastim and L-G-CSF biosimilar were best G-CSF drugs in reducing BP (cumulative probabilities: 20%, 14%, 8%, 8%, respectively). Mecapegfilgrastim, lipegfilgrastim and balugrastim might be the most appreciate G-CSF drugs with both good efficacy and tolerability when treating cancer patients after cytotoxic chemotherapy."

Journal • Retrospective data • Review • Hematological Disorders • Musculoskeletal Pain • Neutropenia • Oncology • Pain

CLINICAL AND ECONOMIC ASSESSMENT OF THE IMPORTANCE OF SUPPORTIVE THERAPY IN ONCOLOGY (ISPOR-EU 2019) - "...Corresponding CERs were € 10,312.1 (for Empegfilgrastim), € 10,506.7 (Lipegfilgrastim), € 11,025.4 (Filgrastim), € 11,556.8 (Pegfilgrastim), € 13,310.5 (Lenograstim), and € 17,378.6 (CT without FN prophylaxis with G-CSF). The use of G-CSF as a preventive measure for the development of chemotherapy complications is economically viable from CEA and COI perspectives. Although the expansion of FN prophylaxis in cancer patients increases drug costs for the FN prevention, it also reduces the total costs of cancer patient treatment."

Clinical