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Pfizer's hemophilia A gene therapy found superior to current treatment in late-stage trial
The gene therapy meets the main goal of showing non-inferiority compared to the Factor VIII replacement therapy in reducing the annual bleeding rate in patients
FILE PHOTO: A compagny logo is seen at a Pfizer office in Puurs, Belgium, December 2, 2022. REUTERS/Johanna Geron/File Photo
Pfizer said on Wednesday that its gene therapy for hemophilia A significantly cut the number of annual bleeding episodes in patients with the rare disorder in a late-stage study and performed better than the current standard treatment.
The strong data takes Pfizer one step closer to securing US regulatory approval for a second gene therapy to treat hemophilia.
Pfizer had received the Food and Drug Administration's nod for Beqvez as a one-time gene therapy for hemophilia B in April, and is priced at $3.5 million in the United States.
Pfizer said its gene therapy for hemophilia A not only met the main goal of showing non-inferiority compared to the Factor VIII replacement therapy in reducing the annual bleeding rate in patients, but also showed superiority to the current standard treatment.
Currently, patients have to go for routine replacement of the clotting protein called Factor VIII (FVIII).
People with hemophilia have a fault in a gene that regulates production of proteins called clotting factors, leading to spontaneous as well as severe bleeding following injuries or surgery. The disorder predominately affects males.
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