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BioMarin All Set For Hemophilia Gene Therapy Approval, But Is It Overestimating Demand?

Gearing Up For August Approval

 
• By Andrew McConaghie

Analysts forecast a slow climb towards peak sales of $2bn – but competitors could erode that figure.

Biomarin
BioMarin needs to persuade payers that a $2-3m price tag for gene therapy is good value compared to conventional therapy • Source: BioMarin

The first ever gene therapy for hemophilia A looks set to gain US approval by August – but just how many patients will receive is one of the key questions hanging over its developer BioMarin Pharmaceutical Inc..

The AAV gene therapy, valoctocogene roxaparvovec (valrox), could transform the lives of many patients with severe hemophilia by freeing them from regular injections of Factor VIII replacement therapy,

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