A groundbreaking clinical trial has demonstrated the efficacy of efanesoctocog alfa in treating children with severe hemophilia A. The study aimed to evaluate the safety of efanesoctocog alfa for bleed prevention in patients under 12, assess the presence of antibodies developed while taking it, and determine whether these antibodies affected the drug’s efficacy.

Over the year-long trial, researchers found no evidence of neutralizing antibodies to FVIII, inhibitors, or anti-drug antibodies. These results confirm that weekly administration of efanesoctocog alfa is a highly effective method to protect children with hemophilia A against bleeding.

Following this successful trial, the FDA has approved efanesoctocog alfa for the treatment of children with severe hemophilia A, marking a significant advancement in pediatric care for this condition.

About the Investigator

Dr. Lynn Malec, MD, MSc
Senior Medical Director
Versiti Comprehensive Center for Bleeding Disorders
Associate Investigator
Versiti Blood Research Institute