Steven Pipe, MD: Novel Hemophilia A Therapy Offers Unprecedented Bleed Protection

At the end of August, investigational therapy efanesoctocog alfa received priority review status from the FDA based on Sanofi's Biologics License Application (BLA) submission for the treatment of hemophilia A.

Steven Pipe, MD, Professor of Pediatrics and Pathology, and Director of the Hemophilia and Coagulation Disorders Program, University of Michigan, explained the magnitude of the results that the phase 3 XTEND-1 trial yielded. According to the data, not only does the novel recombinant factor VIII therapy only require weekly intravenous infusion, it also appeared to be more effective at maintaining healthy factor levels in patients, offering protection from bleeds.

Pipe offered context by acknowledging that, generally, patients who depend on factory prophylaxis often have to do IV infusions at least 3 times a week. With efanesoctocog alfa, patients are able to maintain healthy factor levels for almost a week with a single infusion.

"We've never really had a therapy that would afford such a low burdensome treatment that provides Factor VIII replacement," he said. "But more importantly, we've never had any prophylactic therapy that maintains patients at this level of hemostatic protection well into the normal range for the majority of the week, and I think that's what's really got people excited about this innovation."

The prevention of bleeding events is the primary goal of managing severe hemophilia because the consequences of a bleed can lead to inflammation and damage that extends into risk of joint disease in adulthood.

"If you think about having bleeding events every year, every decade, this is how we accumulate joint disease over time," Pipe said. "So, we're looking for innovative treatments that are going to take us to a new tier of protection from bleeding, and then protection from joint disease."