U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems.
‘s gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems.
Gene therapy developers have typically justified their prices by arguing that the treatments will ultimately lower health care costs by reducing the need for repeat procedures and care over many years. Dr. Margaret Ragni called Roctavian “a major improvement in terms of reducing the burden of disease.” But she notes that many patients are comfortable with their current treatments and may be hesitant to try a new gene therapy.
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