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Pfizer's hemophilia A gene therapy found superior to current treatment in late-stage trial
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Pfizer's hemophilia A gene therapy found superior to current treatment in late-stage trial
Jul 24, 2024 4:14 AM

July 24 (Reuters) - Pfizer ( PFE ) 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 ( PFE ) one step closer to securing

U.S. regulatory approval for a second gene therapy to treat

hemophilia.

Pfizer ( PFE ) 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 ( PFE ) 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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