Margaret Ragni, MD, MPH: Impact of new Antihemophilic Factor VIII Therapy on Hemophilia A

In an interview with HCPLive, Margaret V. Ragni, MD, MPH, Professor of Medicine and Clinical Translational Science, and Medical Director, Hemophilia Center of Western Pennsylvania, discussed the once-weekly antihemophilic factor (recombinant), Fc-VWF-XTEN Fusion Protein-ehtl (ALTUVIIIO) treatment for hemophilia A (previously known as efanesoctocog alfa) that was recently approved by the US FDA for children and adults with the rare condition.

The high sustained factor VIII replacement therapy is indicated for routine prophylaxis, on-demand treatment to control bleeding episodes, and perioperative management.

The approval was based on data from the pivotal XTEND-1 phase 3 study, which showed the therapy had superior efficacy and reduced the treatment burden to once-a-week.

“It's admirable that this study not only looked at bleeds and all the side effects, which there were very few that were important,” Ragni explained, “but they also looked at pain, they looked at quality of life, because those are critical issues for this population.”

After a year of treatment, results showed that the patients’ pain was significantly reduced and their quality of life significantly increased.

“As many as 70% (of patients) had no breakthrough bleeds–that's reportable–if nothing else,” she said. “The drug seems like a win-win to me–time will tell, patients will tell us.”