The Institute for Clinical and Economic Review (ICER) plans to assess the comparative clinical effectiveness and value of Biomarin Pharmaceutical Inc.’s valoctocogene roxaparvovec, an investigational gene therapy being considered for the treatment of hemophilia A. The FDA is expected to make an approval decision on valoctocogene roxaparvovec by midyear. ICER said its analysis likely will compare San Rafael, Calif.-based Biomarin’s therapy with factor VIII replacement therapy and Genentech Inc.’s Hemlibra (emicizumab). ICER opened a comment period Monday that will continue through Jan. 22 to give stakeholders an opportunity to share key information relevant to the development of the evidence report, which will be presented at the August meeting of the New England Comparative Effectiveness Public Advisory Council. 

No Comments