BioMarin announces data from follow up from Phase 3 GENEr8-1 study of ROCTAVIAN

BioMarin Pharmaceutical announced positive results from more than three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIAN, an investigational one-time gene therapy for the treatment of adults with severe hemophilia A. This is the largest and longest global Phase 3 study to date for any gene therapy in hemophilia with 134 participants. In response to the U.S. Food and Drug Administration’s request, and consistent with the FDA’s guidance for other gene therapy trials for hemophilia, BioMarin has also analyzed annualized bleeding rate for all bleeds, regardless of whether those bleeds were treated with exogenous Factor VIII replacement. Results from that analysis were similar to those reflected in the table above. In Year 3, the mean/median ABR for all bleeds was 1.4/0.0, and in Year 4 the mean/median ABR for all bleeds was 1.6/1.0. At the end of Year 3, 92% of patients remained off prophylaxis. Those patients who returned to Factor VIII or emicizumab prophylaxis did so safely. BioMarin plans to present additional data from this study at upcoming medical meetings. As the company has previously indicated, BioMarin is targeting outcomes-based agreements with the three largest health insurance groups that represent about 80% of German lives. The company has executed an OBA with one of the three. The company expects to sign additional agreements in the coming weeks in Germany and continues to progress the European launch of ROCTAVIAN on a country-by-country basis, including meetings with authorities in France and the submission of the reimbursement dossier in Italy. For covered patients, the agreements provide for companion diagnostic testing and reimbursement of ROCTAVIAN, allowing physicians to prescribe and patients to be treated with therapy. The OBAs in Germany are multiyear agreements that cover payer risk of patients potentially returning to prophylaxis through direct BioMarin financial commitment in return for substantial and full upfront payment. Overall, to date, a single 6e13 vg/kg dose of valoctocogene roxaparvovec has been well tolerated with no delayed-onset treatment related adverse events. In Year 3, no new treatment-related serious adverse events or Grade 3 events attributed to valoctocogene roxaparvovec or corticosteroid use emerged. No participants have developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with valoctocogene roxaparvovec. These data will be shared with the FDA as part of the agency’s ongoing review of the Biologics License Application of ROCTAVIAN. The PDUFA date is March 31, 2023, subject to possible agency extension. Additionally, the FDA completed a Pre-License Inspection of the manufacturing facility in early December. BioMarin has provided responses to the comments and observations received at the close of the inspection, and the company believes all are addressable.