JHVEPhoto/iStock Editorial via Getty Images
Sanofi (NASDAQ:SNY) said its hemophilia A therapy, Altuviiio, met the primary safety endpoint in a phase 3 trial.
Altuviiio FVIII therapy was approved last week by the US Food and Drug Administration (FDA) for routine prevention, upon request. treatment and control of bleeding episodes, and perioperative management of bleeding in adults and children.
Hemophilia A is a genetic bleeding disorder caused by insufficient levels of a protein in the blood called factor VIII, which helps blood clot.
The Phase 3 study, called XTEND-Kids, evaluated Altuviiio as a once-weekly preventive therapy in previously treated patients <12 years of age with severe hemophilia A.
The trial met its primary safety endpoint, as no FVIII inhibitors were detected in 74 children, and more than 50 children experienced at least 50 exposure days, nearly a full year of treatment.
The French pharmaceutical giant noted that the completion of the trial represented the final milestone required for regulatory submission in the EU.
The XTEND clinical program consists of two Phase 3 trials in hemophilia A: XTEND-1 in people 12 years of age and older and XTEND-Kids in children less than 12 years of age.
The company added that there is also an ongoing extension study (XTEND-ed).
