BRIEF—ICER to assess gene therapy for hemophilia A

US health technology assessor The Institute for Clinical and Economic Review ICER) announced on Friday that it plans to assess the comparative clinical effectiveness and value of US biotech firm BioMarin Pharmaceutical’s valoctocogene roxaparvovec for the treatment of hemophilia.

Valoctocogene roxaparvovec is an investigational gene therapy, and a decision regarding US Food and Drug Administration approval is expected by mid-2020.

If approved, valoctocogene roxaparvovec – which is also under review with the European Medicines Agency - would be the first gene therapy for any type of hemophilia.

The ICER’s analysis will likely compare valoctocogene roxaparvovec to factor VIII replacement therapy and emicizumab (Hemlibra, from Roche subsidiary Genentech).

The ICER’s Evidence Report on hemophilia A will be reviewed during a public meeting of the New England Comparative Effectiveness Public Advisory Council ( New England CEPAC) in August 2020.