BioMarin Pharmaceutical recently announced that they plan to submit marketing applications to both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec, the company’s investigational gene therapy for the treatment of adults with hemophilia A.
Valoctocogene roxaparvovec uses adeno-associated viruses (AAVs) as vectors to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. AAVs are designed to deliver the genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.
According to a BioMarin press release the submissions are based on results from three years of clinical study data, including a phase 1/2 trial in which adults with hemophilia A experienced significant improvements in annualized bleeding rate (ABR) and requisite FVIII replacement product usage. Access NHF’s recent update on valoctocogene roxaparvovec, to learn more about these trials.
“People with severe hemophilia A continue to experience clinically relevant breakthrough bleeds despite the current standard of care and can be limited in their physical activities,” said Professor John Pasi, MB, ChB, PhD, from Barts and the London School of Medicine and Dentistry; the chief investigator for the valoctocogene roxaparvovec Phase 1/2 study and a principal investigator for the Phase 3 study. “Valoctocogene roxaparvovec represents a potentially transformative investigative therapy that could improve patients’ quality of life, including consequences of bleeding, physical functioning, role functioning, emotional impact, treatment concern, and worry.”
BioMarin plans to submit their marketing applications to the EMA and FDA in fourth quarter of 2019.
Source: BioMarin press release dated July 8, 2019