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Pfizer's gene therapy cuts hemophilia A bleeding rate in late-stage trial
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Pfizer's gene therapy cuts hemophilia A bleeding rate in late-stage trial
Jul 24, 2024 5:06 AM

July 24 (Reuters) - Pfizer's ( PFE ) 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 company said on Wednesday.

The 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, a less

common type of the disorder, in April.

If approved, Pfizer's ( PFE ) hemophilia A gene therapy will compete

with BioMarin Pharmaceutical's one-time treatment Roctavian,

which was approved in the U.S. last year and is priced at $2.9

million.

Currently, the standard-of-care treatment for hemophilia

A patients is to undergo routine replacement of the clotting

protein called Factor VIII.

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.

Data from the 75-patient pool also showed that 84% of

patients who were given Pfizer's ( PFE ) gene therapy had Factor VIII

levels above 5% at 15 months post-infusion, the company said.

The company will present additional data at upcoming medical

meetings, it added. Pfizer ( PFE ) is co-developing the hemophilia A

gene therapy from with Sangamo Therapeutics ( SGMO ), from whom

it had licensed the therapy.

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.

Hemophilia A affects about 25 in every 100,000 male births

worldwide, according to data cited by Pfizer ( PFE ), with majority of

them having a moderate to severe form of the disease for which

the gene therapy was tested.

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