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- Mean Annualized Bleed Rate Reduced by 80% from Baseline and Factor VIII Usage Reduced by 94% in Year 3 Compared to Baseline
- 92% of Patients off Prophylaxis at the End of Year 3
- First Outcomes-Based Agreement (OBA) Recently Signed in Germany; Additional Agreements Expected to be Signed in Coming Weeks
- U.S. Food and Drug Administration Pre-License Inspection of Manufacturing Facility Complete
SAN RAFAEL, Calif., Jan. 8, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced positive results from more than three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIANTM (valoctocogene roxaparvovec), 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.
The ROCTAVIAN data are summarized in the following table:
Phase 3 (6e13 vg/kg dose) | |||
In Year 3* | In Year 4** | ||
FVIII Activity (chromogenic) |
Mean | 18.8 | 15.2 |
Median
| 8.4 | 7.4 | |
Annualized Bleeding Rate*** (bleeding episodes per year) |
Mean | 1.0 | 0.8 |
Median
| 0.0 | 0.0 | |
Annualized FVIII Utilization (infusions per year) |
Mean | 8.4 | 11.1 |
Median
| 0.0 | 0.0 | |
*N=132 (FVIII Activity); N=112 (ABR and AFR). Two of these patients discontinued from the study prior to reaching Year 3. FVIII imputed to be 0 IU/dL; no imputation was carried out for ABR and AFR. **N=17. One of these patients discontinued from the study prior to reaching Year 4. FVIII activity imputed to be 0 IU/dL; no imputation was carried out for ABR and AFR. ***Annualized rate is for treated bleeds. | |||
P values for all primary and secondary endpoint comparisons were
