Hemophilia Seen As Good Testing Ground For Commercializing Gene Therapy
Executive Summary
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.
You may also be interested in...
Roche $4.8bn Buy Sparks Hemophilia Gene Therapy Race
The Swiss major's proposed acquisition of Spark Therapeutics gives it a leading seat at the table in the emerging field of gene therapy.
BioMarin CEO Suggests Hemophilia Gene Therapy Pricing In $2m-$3m Range
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.
Roche's Hemlibra Wins Expanded FDA Approval, Opening The Door To Broad Hemophilia A Opportunity
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.