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.