Lynn Malec, MD: Efanesoctocog alfa Reduces Bleeds, Treatment Burden for Hemophilia

The late-breaking data presented at the annual meeting of the International Society on Thrombosis and Hemostasis has unveiled significant advancements in the treatment of severe hemophilia A in children. Efanesoctocog alfa "efa" (Altuviiio) has emerged as a groundbreaking therapy for pediatric patients with hemophilia A.

"The very exciting piece for this as a therapy is that it offers just once weekly intravenous (IV) dosing, as compared to our other therapies," Lynn Malec, MD, MSc said in an interview. "It allows for patients to have less treatment burden, and it's the only one that's recommended just once weekly."

Lynn Malec, MD, MSc, is the medical director of the Comprehensive Center for Bleeding Disorders, an associate investigator at The Versiti Blood Research Institute, and associate professor of medicine & pediatrics at the Medical College of Wisconsin.

"Also importantly, is patients actually have high sustained factor VIII (FVIII) activities really throughout the course of the week," she continued. "This was something that was seen in this under 12 (years of age) cohort, as it had been seen in older adolescents and adults that have been previously published."

Efa earned the distinction of being the first and only FVIII therapy to receive Breakthrough Therapy designation from the US Food and Drug Administration (FDA), emphasizing the drug's potential to address unmet needs within the hemophilia community.

The FDA recognizes efa as a "first and best-in-class high-sustained FVIII therapy," representing a new class of treatment options for pediatric and adult patients with severe hemophilia A.

The XTEND-1 phase 3 study formed the basis for efa's Breakthrough Therapy designation by demonstrating the clinically meaningful prevention of bleeds and the superiority of efa compared with prior prophylaxis factor treatments in terms of preventing bleeding episodes.

In addition to bleed prevention, the study evaluated secondary endpoints, including pharmacokinetics, treatment response for bleeding events, and outcomes for patients undergoing surgeries.