US drug maker Pfizer Inc said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study.
Data from the study showed that a single dose of the therapy was superior to the current standard of care in helping reduce the bleeding rate in patients with moderately severe to severe forms of hemophilia B.
The disorder hampers the body's ability to make a blood-clotting protein called factor IX.
Pfizer's therapy, fidanacogene elaparvovec, is designed to help patients produce factor IX themselves after a one-time treatment, as opposed to current treatments, which focus on regular infusions of the protein.