Lynn Malec, MD: FVIII Therapy Improves Levels in Pediatric Patients with Hemophilia A

Recent data on efanesoctocog alfa "efa" (Altuviiio) demonstrated promising results in pediatric patients with hemophilia A. Patients were eligible for the open label XTEND-1 phase 3 study if they were < 12 years of age and had severe hemophilia A.

"The patient population had been what we call previously treated patients or PTPs, and those patients that were between 6 and < 12 years of age had received at least 150 prior exposure days that they had received a factor product," Lynn Malec, MD, MSc, said in an interview with HCPLive. "For the patients that were < 6, they needed at least 50 exposure days and then they received weekly dosing at 50 units per kilo, once a week, over the course of 52 weeks of study."

Malec, the medical director of the Comprehensive Center for Bleeding Disorders, associate investigator at The Versiti Blood Research Institute, and associate professor of medicine & pediatrics at the Medical College of Wisconsin, explained that efa has a high sustained factory therapy designation.

"Patients had a factor VIII level that was above 40% for approximately 3 days, above 15% for approximately 5 days, and then above 10%, for that week," she said. "That's really a substantially different kind of (pharmacokinectic) curve, or factor VIII, over the course of the week."

The therapy was the first and only FVIII to be awarded Breakthrough Therapy designation by the US Food and Drug Administration (FDA) and defined a new therapy class by the agency as the first and best-in-class high-sustained FVIII therapy.