Fidanacogene Elaparvovec for Hemophilia B — A Multiyear Follow-up Study
Fidanacogene elaparvovec was associated with no or only low-grade adverse effects over a period of 3 to 6 years. Efficacy was maintained in the long term at 5×10<sup>11</sup> vg per kilogram, one of the lowest intravenous doses of AAV used for any indication. (Funded by Pfizer; ClinicalTrials.gov number, NCT03307980.).
Authors
- Jerome Teitel (ORCID: https://orcid.org/0000-0001-6455-8167)
- Ian Winburn
- Amit Chhabra (ORCID: https://orcid.org/0000-0002-6115-8253)
- Amanda C. O’Brien
- Ype P. de Jong (ORCID: https://orcid.org/0000-0001-6357-2370)
- John E.J. Rasko (ORCID: https://orcid.org/0000-0003-2975-807X)
- Lynne M. Smith (ORCID: https://orcid.org/0000-0002-3086-9167)
- Lindsey A. George (ORCID: https://orcid.org/0000-0002-9763-1559)
- Jonathan M. Ducore (ORCID: https://orcid.org/0000-0003-4282-0632)
- Catherine McGuinn (ORCID: https://orcid.org/0000-0002-3421-0925)
- Jeremy W. Rupon (ORCID: https://orcid.org/0000-0001-8751-3834)
- Adam Giermasz
- Benjamin J. Samelson‐Jones (ORCID: https://orcid.org/0000-0001-6772-4140)
- Katherine A. High
Institutions
- The University of Sydney (AU)
- St. Michael's Hospital (CA)
- Children's Hospital of Philadelphia (US)
- Pfizer (United States) (US)
- University of Toronto (CA)
- Pfizer (United Kingdom) (GB)
- Cornell University (US)
- Royal Prince Alfred Hospital (AU)
- Weill Cornell Medicine (US)
- University of Pennsylvania (US)
- University of California, Davis (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-04-16
- DOI
- https://doi.org/10.1056/nejmoa2307159
- Citations
- 14
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 24.78
Funders
- Pfizer