U.S. health regulators have approved a gene therapy for the most common form of hemophilia
This photo provided by BioMarin in June 2023 shows a vial and packaging for their drug Roctavian. U.S. officials on Thursday, June 19, 2023 approved the drugmaker's gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems. WASHINGTON — — U.S.
BioMarin did not immediately announce the price. A similar gene therapy approved last year for hemophilia BGene 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. The San Rafael, California-based company excluded patients with certain potentially complicating conditions, including liver disorders and resistance to the standard blood clotting protein, which often develops in some hemophilia patients. BioMarin’s president for research and development, Dr. Henry Fuchs, said the company is conducting studies in some of those excluded groups to see if they can safely receive the therapy.
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