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Stock impact report

FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world [CNN]

GoodRx Holdings, Inc. - Class A (GDRX) 
Company Research Source: CNN
Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which people do not produce a protein needed to create blood clots. About 1 in 40,000 people have the disease, most of whom are men. Typical treatment requires routine intravenous infusions to maintain sufficient levels of the missing or deficient clotting factor, but the new gene therapy is meant to be a one-time IV infusion. “Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals' quality of life,” Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a statement . “Today's approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this for Show less Read more
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