Valoctocogene roxaparvovec (BioMarin), also known as valrox, is an adeno-associated virus-mediated gene therapy that delivers human B domain-deleted coagulation factor VIII (fVIII) to patients with hemophilia A. The gene therapy is being developed by BioMarin, which initiated its pivotal Phase III program in December 2017. Valoctocogene roxaparvovec has been granted expedited review designations in both the US and EU, and represents the most advanced gene therapy for any type of hemophilia currently in the pipeline.
Data from Phase II showed a strong reduction in the number of bleeding episodes, albeit with a wide range of fVIII expression levels. The wide variability between patients may allow for spontaneous bleeding episodes in those with lower fVIII expression, while extremely high levels can result in thromboembolic events. The challenge for BioMarin in Phase III will be to create a predictable and stable response to treatment, identifying the correct dose for each patient.
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