Introduction: Etranacogene dezaparvovec is the first gene therapy approved for treatment of adults with severe and moderately severe hemophilia B.
Areas Covered: This review describes the results of the clinical trial program of AMT-060 and etranacogene dezaparvovec, outlining the pharmacokinetic, clinical efficacy and safety data. With the entry of etranacogene dezaparvovec into the market, this review summarizes the treatment landscape in hemophilia B and discusses the current unknowns in the field.
Expert Opinion: Gene therapy appears to be a feasible option for adults with severe and moderately severe hemophilia B. Etranacogene dezaparvovec enables most patients to reach stable factor IX (FIX) levels after a single intravenous infusion, eliminating the need for regular prophylaxis; thus, drastically reducing treatment burden and avoiding variable bleeding risk owing to fluctuating FIX activity levels. Efficacy of etranacogene dezaparvovec has been demonstrated even in the presence of preexisting neutralizing antibodies (up to a titer of 1:678), with a relative low risk of transaminitis and its associated potential loss of transgene expression. However, long-term data are required to ascertain the durability of FIX levels achieved and safety. The cost-effectiveness and adoption of innovative payment models for reimbursement are key in choosing gene therapy over existing treatments.
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Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA.
Appl Health Econ Health Policy
December 2024
Department of Pharmacy Systems, Outcomes, and Policy, Retzky College of Pharmacy, University of Illinois Chicago, Chicago, IL, USA.
Article Synopsis
- - The study evaluates the cost-effectiveness of a new gene therapy, Etranacogene dezaparvovec (EDZ), for severe hemophilia B, comparing it to the traditional treatment, factor IX (FIX) prophylaxis over a lifetime perspective in the U.S.
- - Results show that despite EDZ costing $3.5 million, it offers lifetime savings of $11 million and slightly improves quality of life by 0.64 QALYs compared to FIX, which has very high annual costs.
- - The cost-effectiveness of EDZ is influenced significantly by capping annual cost offsets, emphasizing how these limits can help balance price and value for healthcare systems.
Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.
Pharmacoeconomics
January 2025
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands.
Objective: To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.
Methods: A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population.
Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B.
J Thromb Haemost
January 2025
Department of Hematology, Rigshospitalet, Copenhagen, Denmark.
Background: Little information regarding the management of invasive procedures in people with hemophilia B (HB) after undergoing gene therapy is available. Here, we report the management of invasive procedures in people with severe or moderately severe HB who had previously been treated with etranacogene dezaparvovec in the phase 2b and phase 3 Health Outcomes with Padua Gene; Evaluation in Hemophilia B clinical trials (NCT03489291 and NCT03569891).
Objectives: The objective of this study was to describe the use of exogenous FIX, endogenous FIX activity prior to invasive procedures, and peri- and postoperative bleeds in participants who underwent invasive procedures after receiving etranacogene dezaparvovec gene therapy.
Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.
Haemophilia
November 2024
Àrea del medicament, Institut Català de la Salut, Barcelona, Spain.
Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.
Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).
Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria.
Biological Barriers for Drug Delivery and Development of Innovative Therapeutic Approaches in HIV, Pancreatic Cancer, and Hemophilia A/B.
Pharmaceutics
September 2024
WIR-Walk In Ruhr, Center for Sexual Health & Medicine, Department of Dermatology, Venerology and Allergology, Ruhr-University Bochum, 44787 Bochum, Germany.
Article Synopsis
- Biological barriers pose significant challenges in the development of new therapies, as drugs must navigate various protective layers at both the tissue and cellular levels to effectively target specific cells.
- A diverse range of therapeutic options is now available, from small molecules to advanced techniques like gene therapies and monoclonal antibodies, with recent innovations rapidly transforming treatment approaches.
- Notable advancements, such as the FDA approval of new RNA-based therapies and viral-vector gene therapies for hemophilia, exemplify the potential for innovative medicines to address complex diseases and improve patient care.
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