The Food and Drug Administration on Thursday approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder. Here's what you need to know:
, according to data presented to regulators. It’s also the latest FDA-approved gene therapy, heralding what some scientists say is the future of treating thousands of diseases.sickle cell anemia and spinal muscular atrophy.
Use of the technology is relatively rare, in part because gene therapies are among the most expensive drugs in the world. Roctavian will cost $2.9 million for a single infusion; Hemgenix, which treats another form of hemophilia, costs $3.5 million per use,Advertisement
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