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.