Data from Longest and Largest Hemophilia Gene Therapy Study Show Durable and Sustained Bleed Control and Factor VIII Expression Maintained Four Years Post-ROCTAVIAN Infusion. Additional Data Show Meaningful Impact of ROCTAVIAN on Health-Related Quality of Life (HRQoL).
SAN RAFAEL, Calif., June 7, 2024 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that new data supporting the long-term safety and efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, June 22-26, 2024.
The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained four years after treatment with ROCTAVIAN, with FVIII activity near stable compared with results reported previously and no new safety signals observed. Of the 134 patients who received ROCTAVIAN in the study, the rollover population of 112 patients had baseline annualized bleeding rate (ABR) data prospectively collected during a period of at least six months while on routine FVIII prophylaxis prior to receiving ROCTAVIAN, and two of the 112 patients discontinued the study prior to year four. During year four, 73.6% of the remaining participants (81/110) had zero treated bleeds. Over the entire study period to the time of the data cut, 24 of the 134 total participants resumed prophylaxis with either FVIII or emicizumab without any complications. Mean FVIII activity at the end of year four (n=130) was 27.1 and 16.1 IU/dL as assessed by one-stage assay (OSA) and chromogenic assay (CSA), respectively. These levels are near stable from the previously reported three-year data. Over four years, the mean ABR for treated bleeds for the rollover population was 0.8 bleeds/year, and the mean ABR for all bleeds was 1.3 bleeds/year.
