Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Johnny MahlanguRadoslaw KaczmarekAnnette von DrygalskiSusan ShapiroSheng-Chieh ChouMargareth C OzeloGili KenetFlora PeyvandiMichael WangBella MadanNigel S KeyMichael LaffanAmy L DunnJane MasonDoris V QuonEmily SymingtonAndrew D LeavittJohannes OldenburgHervé ChambostMark T RedingKala JayaramHua YuReena MahajanKonstantia-Maria ChaveleDivya B ReddyJoshua HenshawTara M RobinsonWing Yen WongSteven W Pipenull nullPublished in: The New England journal of medicine (2023)
The study data show the durability of factor VIII activity and bleeding reduction and the safety profile of valoctocogene roxaparvovec at least 2 years after the gene transfer. Models of the risk of joint bleeding suggest that the relationship between transgene-derived factor VIII activity and bleeding episodes is similar to that reported with the use of epidemiologic data for persons with mild-to-moderate hemophilia A. (Funded by BioMarin Pharmaceutical; GENEr8-1 ClinicalTrials.gov number, NCT03370913.).