Hemgenix (etranacogene dezaparvovec-drlb) / uniQure, CSL Behring - LARVOL DELTA

Concizumab (Alhemo) for hemophilia A and B with inhibitors. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients (clinicaltrials.gov) - P3 | N=67 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

A Targeted Literature Review of Value-Based Agreements (VBAs) for Cell and Gene Therapies in the United States (ISPOR 2025) - "The therapies with VBAs in place included: Beqvez, Casgevy, Hemgenix, Kymriah, Luxturna, Lyfgenia, Roctavian, Vyjuvek, Zolgensma, and Zynteglo... In this review, multiple VBAs for CGTs were identified across multiple disease areas. Most payers did not publicly disclose which outcomes measures the VBAs were assessing. Of those that did, outcomes assessed could be sourced from routine patient visits and/or adjudicated claims, placing no additional burden on providers to collect data for the sole purpose of the VBA."

Gene therapy • Review • Gene Therapies

Do Breakthrough Therapies Demonstrate Breakthrough Value to Payers? An Evaluation of US Hemophilia B Gene Therapy Access Policies (ISPOR 2025) - "US payer policies defined a narrow patient population for Hemophilia B gene therapies compared to product FDA labels, sometimes restricting eligibility beyond clinical trial criteria. This targeted review identified misalignment between payers and regulators on the clinical value of Hemophilia B Breakthroughs."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

GENE THERAPY OF HEMOPHILIA B: IDENTIFICATION OF THE MOST EFFICIENT AAV SEROTYPE VECTOR FOR TRANSDUCTION OF HUMAN HEPATIC CELLS (ASGCT 2025) - "In 2022, the United States Food and Drug Administration (USFDA) approved Hemgenix, an AAV5-based vector from uniQure for gene therapy of hemophilia B. A relative high dose of 2x1013 vgs/kg of this vector leads to up to ~12% expression of the human clotting factor IX (hFIX)...All of these proof-of-concept studies make a compelling case for the use of AAV3 vectors with lower immunogenicity, improved safety, ensuring translation to the clinic with higher probability of success for gene therapy of hemophilia B. Furthermore, the reduced vector production costs as well as lower cost per patient should allow the eligible patient population worldwide to benefit from AAV3 vector-mediated gene therapy of hemophilia B in particular, and human liver diseases in general, in which other AAV serotype vectors have proven to be less than optimal. Disease Focus of Abstract:Hemophilia"

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatology • Rare Diseases • MET

A Novel Agent-based Computational Model for Liver-targeting, AAV-based Gene Therapies Could Predict Response Durability in Hemophilia B Patients Treated with Etranacogene Dezaparvovec (ASGCT 2025) - P3 | "While the model was applied here in the context of treating hemophilia B, the modeling and simulation-based framework should be adaptable to many other AAV-based gene therapies. Disease Focus of Abstract:Hemophilia"

Clinical • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

First Patient Treated with HEMGENIX (etranacogene dezaparvovec) Gene Therapy for Haemophilia B in Austria (CSL Behring Press Release) - "CSL Behring Austria today announced that the first haemophilia B patient in Austria was treated with the gene therapy HEMGENIX (etranacogene dezaparvovec) at the Comprehensive Care Center of the University Hospital of Medicine Vienna....HEMGENIX is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors."

Launch Europe • Hemophilia B

Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. (PubMed, Blood Adv) - P, P2 | "Participants are eligible to participate in an extension study (NCT05962398) for 10-years additional follow-up. ClinicalTrials.gov Identifier: NCT03489291."

Journal • P2b data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Gene therapy as an innovative approach to the treatment of hemophilia B-a review. (PubMed, J Appl Genet) - "An innovative proposal that, despite initial concerns, is becoming more and more popular every day is the recently approved genetic therapy in Europe, which uses viral vectors to transfer the correct gene that encodes coagulation factor IX. The introduction of a recombinant gene in place of its defective counterpart seems to be a promising solution and the beginning of a new era in which genetic therapies have a chance to develop their full potential and replace existing therapeutic regimens."

Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Germany will reimburse haemophilia gene therapy Hemgenix (pharmaphorum) - "CSL Behring has added another major European market to the list of those providing reimbursement coverage of its haemophilia B gene therapy Hemgenix after agreeing an access deal in Germany...The agreement with the GKV-Spitzenverband – the national association of statutory health insurance funds – means that Hemgenix (etranacogene dezaparvovec) can now be reimbursed across Germany's health system as a treatment for severe and moderately severe haemophilia B in adult patients without a history of Factor IX (FIX) inhibitors....In Germany, the gene therapy is said to be the first covered under a national, success-based reimbursement model, with the cost of treatment – which is upwards of €3 million ($3.3 million) in EU countries – tied to its success in each individual patient."

Reimbursement • Hemophilia B

The deLIVERed promises of gene therapy: past, present and future of liver-directed gene therapy. (PubMed, Mol Ther) - "Adeno-associated virus (AAV) vectors have become the cornerstone of liver-directed therapies, demonstrating remarkable success in conditions such as hemophilia A and B, with FDA approved therapies like Etranacogene Dezaparvovec, Beqvez, and Roctavian marking milestones in the field. The advent of various genome-editing tools to repair genomic mutations or insert therapeutic DNAs into precise locations in the genome further enhances the potential for a single-dose medicine that will offer durable life-long therapeutic treatments. As advancements accelerate, liver-targeted gene therapy is poised to continue to transform the treatment landscape for both genetic and acquired disorders, for which unmet challenges remain."

Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Hepatology • Immune Modulation • Immunology • Rare Diseases

The Ministry of Health publishes the first reports that will justify the financing of drugs [Google translation] (Infobae) - "The Ministry of Health began publishing reports on Friday justifying the criteria used to decide to fund a drug in order to improve transparency regarding the process of including medicines in the public system...The first three drugs that already have their respective reports posted on the website are etranacogene dezaparvovec (Hemgenix, by its commercial name, used for hemophilia B); mavacamten (Camzyos, indicated for hypertrophic obstructive cardiomyopathy) and etrasimod (Velsipity, for ulcerative colitis)...Thus, from now on, each new medicine that is incorporated into the National Health System will have its own file detailing information about its financing conditions or comparative evaluation with similar medicines, the Ministry explained in a note....It also includes information on the decision of the CIPM, the conditions of inclusion, detailing the specific criteria by which the drug was included in the SNS, from the target population to possible restrictions, among others."

Reimbursement • Hemophilia B • Obstructive Hypertrophic Cardiomyopathy • Ulcerative Colitis

The new paradigm for treating hemophilia B in Spain is now a reality: “It is a very important change” [Google translation] (GacetaMedica) - "The innovative gene therapy Hemgenix (etranacogene dezaparvovec, from CSL Behring) is now available in Spain, where the Interministerial Commission on the Price of Medicines (CIPM) granted it public funding last September. It is thus presented as the only gene therapy available to treat severe and moderately severe hemophilia B and also represents a paradigm shift , especially in treatment....Under the motto....at a meeting organized by Gaceta Médica, which had the support of CSL Behring , professionals and patients discussed the challenges and opportunities brought by this new therapy for the hereditary bleeding disorder caused by the deficiency of factor IX, an essential protein for correct blood clotting, which causes affected patients to experience episodes of prolonged bleeding."

Launch Europe • Hemophilia B

CSL Behring’s Gene Therapy HEMGENIX (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B (PRNewswire) - P3 | N=67 | HOPE-B (NCT03569891) | Sponsor: CSL Behring | "94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment; Mean factor IX activity levels were sustained at near normal levels of 37% through four years post-treatment, reinforcing the efficacy of HEMGENIX in the treatment of hemophilia B...HEMGENIX produced mean factor IX levels of 41.5 IU/dL (n=50) at year one, 36.7 IU/dL (n=50) at year two, 38.6 IU/dL (n=48) at year three and 37.4 IU/dL (n=47) at year four post-infusion. In addition, mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in (4.16, n=54) as compared to year four (0.40, n=51). Furthermore, joint bleeds were reduced from a mean ABR of 2.34 at lead-in to 0.09 during year four. In year four, 94% of patients remained free of continuous prophylaxis treatment."

P3 data • Hemophilia B

The SFDA Approves the First Gene Therapy for Hemophilia (B) in Saudi Arabia (Saudi Food and Drug Authority (SFDA)) - "The Saudi Food and Drug Authority (SFDA) has approved the first gene therapy for Hemophilia (B) in the Kingdom of Saudi Arabia. It has announced the registration of Hemgenix (etranacogene dezaparvovec) for use in patients with moderate to severe Hemophilia (B), a life-threatening genetic disorder."

Approval • Hemophilia B

Gene-ius at work: Hemophilia B treatment enters a new era. (PubMed, Am J Health Syst Pharm) - "For the first time in history, individuals with hemophilia B have access to potentially curative therapies through gene therapy. Both etranacogene dezaparvovec and fidanacogene elaparvovec offer significant efficacy, reducing the number of bleeding episodes and raising FIX concentrations with a single lifetime administration. While concerns remain regarding long-term safety and durability, these therapies represent a major advancement in reducing treatment burden and improving quality of life for patients. The future of hemophilia B management now holds the promise of greater independence from frequent prophylactic treatments."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Natural history of AAV5 neutralising antibodies in adults with haemophilia B during ≥6-month screening and lead-in to the HOPE-B trial with etranacogene dezaparvovec gene therapy (EAHAD 2025) - No abstract available

Clinical • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Do value perceptions on the contribution of etranacogene dezaparvovec for the treatment of severe and moderately severe Haemophilia B vary between clinicians and hospital pharmacists? A Multicriteria Decision Analysis study (EAHAD 2025) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Four-year results of etranacogene dezaparvovec in haemophilia B patients without AAV5 neutralising antibodies: Phase 3 HOPE-B trial (EAHAD 2025) - No abstract available

Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Four-year results of etranacogene dezaparvovec in haemophilia B patients with pre-existing AAV5 neutralising antibodies: Phase 3 HOPE-B trial (EAHAD 2025) - No abstract available

Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

First patients treated with HEMGENIX (etranacogene dezaparvovec) gene therapy for haemophilia B in Denmark (Mynewsdesk) - "CSL Behring Denmark today announced that the first Danish patients with haemophilia B were treated with the gene therapy HEMGENIX(etranacogene dezaparvovec)....Patients in Denmark can access HEMGENIX through an innovative outcome-based agreement with Amgros, finalised in October 2024. This makes Denmark the first Nordic and European country to adopt a performance-based model, where costs are incurred only as long as the gene therapy proves effective over the agreed long-term period."

Launch Europe • Hemophilia • Hemophilia B

In Vitro and In Vivo Potency Differences between AAVRh74var and AAV5 Vectors Encoding the Same High-Activity Human Factor IX Variant, FIX-R338L, Expression Cassette: Implications for Hemophilia B Gene Therapy (ASH 2024) - "The only other approved HB gene therapy (etranacogene dezaparvovec) utilizes the AAV5 capsid and is administered at a 40-fold higher dose (2×1013 gc/kg). Overall, the results demonstrate differences between the AAVRh74var and AAV5 capsids. This is consistent with fidanacogene elaparvovec (AAVRh74var) achieving clinically robust efficacy, despite a several-fold lower dose than AAV5-based gene therapies."

Gene therapy • Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatocellular Cancer • Oncology • Rare Diseases • Solid Tumor

Improved Joint Health in the Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B over 36 Months of Follow up (ASH 2024) - "Conclusions : A single infusion of etranacogene dezaparvovec resulted in a discontinuation of continuous FIX concentrate prophylaxis, a reduction of (treated) joint ABR, which was significantly related to the steady state FIX level, and improved HJHS, with a positive effect on TJ resolution and prevention. The etranacogene dezaparvovec trial population, despite highly prevalent baseline hemophilic joint damage, demonstrated stable or improved joint health over 36 months of sustained endogenous FIX Padua expression."

Gene therapy • P3 data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Musculoskeletal Pain • Orthopedics • Pain • Rare Diseases • Rheumatology

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B (ASH 2024) - P, P1/2 | "Conclusions : Durability of factor expression is a key consideration in the decision-making process about gene therapy for patients and physicians. With just one amino acid difference in the expressed FIX protein, CSL220 is the precursor of etranacogene dezaparvovec (CSL222), which was the first gene therapy approved for the treatment of hemophilia B. This 8-year follow-up after CSL220 administration provides continued evidence for the durability, stability, and safety of FIX expression after AAV5-based gene therapy for the treatment of hemophilia B."

Clinical • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Development of an Agent-Based Model to Investigate Response Durability in Hemophilia B Patients Treated with the AAV-Based Coagulation Factor IX (FIX) Gene Therapy Etranacogene Dezaparvovec (ASH 2024) - P3 | "This work demonstrates that ABMs can be used to predict how biological variables such as target cell turnover, liver physiology and viability along with product characteristics may impact response durability. While the model is applied herein to the context of AAV gene therapy-based FIX for hemophilia B, the computational framework should be adaptable to many other AAV-based gene therapies and diseases."

Clinical • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases