The United States Federal Drug Administration (FDA) has just approved a new treatment for a rare blood clotting disease, one with a hefty price tag.
Hemgenix, on the other hand, is a one-time intravenous product given in a single dose at a fraction of the price. The product is carried into the body via a, which is engineered to deliver DNA to target cells in the liver. This genetic information is then replicated by cells, spreading the instructions for a clotting protein, known as Factor IX.
After receiving the gene therapy, the rate at which patients developed uncontrolled bleeds fell by over 50 percent compared to their baseline rate. "Gene therapy for hemophilia has been on the horizon for more than two decades.
As part of this designation, the Hemgenix manufacturer, CSL Behring, has exclusive rights to the US marketplace for the next seven years.
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