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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
Open accessAbstract
BACKGROUND: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1-2 dose-escalation study. METHODS: vector genomes of valoctocogene roxaparvovec per kilogram of body weight. The primary end point was the change from baseline in factor VIII activity (measured with a chromogenic substrate assay) during weeks 49 through 52 after infusion. Secondary end points included the change in annualized factor VIII concentrate use and bleeding rates. Safety was assessed as adverse events and laboratory test results. RESULTS: Overall, 134 participants received an infusion and completed more than 51 weeks of follow-up. Among the 132 human immunodeficiency virus-negative participants, the mean factor VIII activity level at weeks 49 through 52 had increased by 41.9 IU per deciliter (95% confidence interval [CI], 34.1 to 49.7; P<0.001; median change, 22.9 IU per deciliter; interquartile range, 10.9 to 61.3). Among the 112 participants enrolled from a prospective noninterventional study, the mean annualized rates of factor VIII concentrate use and treated bleeding after week 4 had decreased after infusion by 98.6% and 83.8%, respectively (P<0.001 for both comparisons). All the participants had at least one adverse event; 22 of 134 (16.4%) reported serious adverse events. Elevations in alanine aminotransferase levels occurred in 115 of 134 participants (85.8%) and were managed with immune suppressants. The other most common adverse events were headache (38.1%), nausea (37.3%), and elevations in aspartate aminotransferase levels (35.1%). No development of factor VIII inhibitors or thrombosis occurred in any of the participants. CONCLUSIONS: In patients with severe hemophilia A, valoctocogene roxaparvovec treatment provided endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis. (Funded by BioMarin Pharmaceutical; GENEr8-1 ClinicalTrials.gov number, NCT03370913.).
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Cites
Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
Self-citationWFH Guidelines for the Management of Hemophilia, 3rd edition
Srivastava, A., Santagostino, E., Dougall, A., Kitchen, S., Sutherland, M., Pipe, S. W., Carcao, M., Mahlangu, J., Ragni, M. V., Windyga, J., et al. (2020) · Haemophilia · doi:10.1111/hae.14046 · Cited by 2,033
Self-citationAdenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864
Self-citationLong-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273
Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors
Mahlangu, J., Oldenburg, J., Paz-Priel, I., Negrier, C., Niggli, M., Mancuso, M. E., Schmitt, C., Jiménez-Yuste, V., Kempton, C., Dhalluin, C., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1803550 · Cited by 763
Self-citationHemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708538 · Cited by 762
Self-citationAAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Rangarajan, S., Walsh, L., Lester, W., Perry, D., Madan, B., Laffan, M., Yu, H., Vettermann, C., Pierce, G. F., Wong, W. Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708483 · Cited by 694
Self-citationAAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Costa Verdera, H., Kuranda, K., & Mingozzi, F. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2019.12.010 · Cited by 641
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 551
Cited by
Adeno-associated virus as a delivery vector for gene therapy of human diseases
Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 904
Self-citationVarious AAV Serotypes and Their Applications in Gene Therapy: An Overview
Issa, S. S., Shaimardanova, A. A., Solovyeva, V. V., & Rizvanov, A. A. (2023) · Cells · doi:10.3390/cells12050785 · Cited by 313
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
Shen, W., Liu, S., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.1001263 · Cited by 173
Butterfield, J. S. S., Yamada, K., Bertolini, T. B., Syed, F., Kumar, S. R. P., Li, X., Arisa, S., Piñeros, A. R., Tapia, A., Rogers, C. A., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.07.005 · Cited by 36
Selection of UTRs in mRNA-Based Gene Therapy and Vaccines
Volkhin, I. A., Paremskaia, A. I., Dashian, M. A., Smeshnova, D. S., Pavlov, R. E., Mityaeva, O. N., Volchkov, P. Y., & Deviatkin, A. A. (2025) · Biochemistry (Moscow) · doi:10.1134/s0006297924604659 · Cited by 7
Metadata
- Authors
- Ozelo, M. C., Mahlangu, J., Pasi, K. J., Giermasz, A., Leavitt, A. D., Laffan, M., Symington, E., Quon, D. V., Wang, J.-D., Peerlinck, K., Pipe, S. W., Madan, B., Key, N. S., Pierce, G. F., O’Mahony, B., Kaczmarek, R., Henshaw, J., Lawal, A., Jayaram, K., Huang, M., Yang, X., Wong, W. Y., & Kim, B.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2022-03-17
- Volume
- 386
- Issue
- 11
- Pages
- 1013-1025
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W4221015126
- Cited by
- 417
- References
- 30
- FWCI
- 54.62
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Ozelo, M. C., Mahlangu, J., Pasi, K. J., Giermasz, A., Leavitt, A. D., Laffan, M., Symington, E., Quon, D. V., Wang, J.-D., Peerlinck, K., Pipe, S. W., Madan, B., Key, N. S., Pierce, G. F., O’Mahony, B., Kaczmarek, R., Henshaw, J., Lawal, A., Jayaram, K., . . . Kim, B. (2022). Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A. New England Journal of Medicine. https://doi.org/10.1056/nejmoa2113708
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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