Hemophilia Seen As Good Testing Ground For Commercializing Gene Therapy
With a well characterized genetic driver, a clear marker to show whether it's working, and available treatments that carry high annual costs, hemophilia may be a good testing ground for commercializing gene therapy, but payers still have to work out how to reimburse the one-time medicines.
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The Swiss major's proposed acquisition of Spark Therapeutics gives it a leading seat at the table in the emerging field of gene therapy.
During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.
Hemlibra was approved to prevent or reduce bleeding in adults and children with hemophilia A without factor VIII inhibitors, a significantly larger patient population than its current indication in patients with factor VIII inhibitors.