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. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, a $2.
BioMarin said Roctavian's $2.9 million price tag reflects"the possibility of freedom from years" of infusions, which cost about $800,000 annually for a typical patient. The price is less than the $3.5 million announced last year for aLike most medicines in the U.S., the new treatment will mainly be paid for by insurers, not patients.
Roctavian uses an inactivated virus, created in a lab, to deliver a replacement gene to the liver cells that produce the clotting protein. When the therapy is successful, patients can then produce the protein themselves. The label warns that rare, severe allergic reactions can occur. BioMarin was among the first companies to begin testing an experimental gene therapy in patients more than six years ago.
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Gene therapy for severe hemophilia is approved by FDAU.S. health regulators have approved a gene therapy for the most common form of hemophilia
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