ICER considers hemophilia drug values against backdrop of costly factor VIII prices

20 October 2020
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The USA's Institute for Clinical and Economic Review (ICER) has released an Evidence Report assessing the comparative clinical effectiveness and value of BioMarin Pharmaceutical’s (Nasdaq: BMRN) Roctavian (valoctocogene roxaparvovec) and Roche’s (ROG: SIX) Hemlibra (emicizumab) for the treatment of hemophilia A.

In August 2020, BioMarin received a Complete Response Letter from the US Food and Drug Administration (FDA) requesting an additional two years of follow-up safety and efficacy data on valoctocogene roxaparvovec, and that process may delay the gene therapy’s approval decision in the USA until at least 2022.

'Possibility of a cure on the horizon'

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