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Efanesoctocog Alfa: The Breakthrough Hemophilia Therapy Candidate

Dr. Steven Pipe talks about the profound effect the novel recombinant factor VIII candidate demonstrated in patients with hemophilia A.

In an interview with HCPLive, Steven Pipe, MD, Professor of Pediatrics and Pathology, and Director of the Hemophilia and Coagulation Disorders Program, University of Michigan, discussed the impact of efanesoctocog alfa on patients with hemophilia A in the phase 3 XTEND-1 clinical trial.

"We've had some tremendous innovation in this space, particularly for hemophilia A, over the last few years, we have had some other extended half life Factor VIII products, but none of them have achieved the kind of pharmacokinetics that we're observing with efa (efanesoctocog alfa)," he explained.

The novel recombinant factor VIII therapy just received priority review status from the FDA at the end of the summer with a decision expected by February 28, 2023. The phase 3 clinical trial data was submitted by developing company Sanofi along with the Biologics License Application (BLA).

Because efanesoctocog alfa is an intravenous therapy that would only require administration once per week, compared with 3 or more times per week, this candidate has the potential to improve treatment adherence, and subsequently, the control of bleeding events and overall patient health.

"The other thing that is important for this type of therapy is the spontaneity that will come back to patients' lives," Pipe said.

Those who live with the rare condition must regularly be monitoring their factor levels, even throughout the day, which contributes to almost every decision they make, he explained. Whether an individual is invited to a friend's for dinner or planning on participating in a family hike, their factor levels need to be within healthy range.

"This type of therapy, maintaining levels for the majority of the week in the normal range is going to return a lot of that spontaneity to patients, and they won't have to think as much about where their plasma levels are at any given time," Pipe said.

He elaborated on the breakthrough potential of efanesoctocog alfa.

"We're really striving to take patients to higher and higher levels of, what we would call, health equity. We want our patients to live lives that they choose, reach the goals that they're shooting for, and to have a level of health equity that is similar to their unaffected peers. And I think this type of innovation is taking us one step closer to achieving all of those goals."