Pfizer Inc. (NYSE:PFE)today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for HYMPAVZI® (marstacimab-hncq) to include the treatment of patients with hemophilia A or B 12 years and older with inhibitors and pediatric patients (ages 6 to 11 years) with or without inhibitors. HYMPAVZI is now indicated in the U.S. for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 6 years of age and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) with or without factor IX inhibitors.

HYMPAVZI offers a combination of prophylactic bleed protection with a straightforward, once-weekly subcutaneous administration that does not require routine treatment-related lab monitoring.

"For children who have to deal with bleeding episodes from an early age and for people living with hemophilia who develop inhibitors, treatment options have been limited and are often burdensome," said Guy Young, M.D., Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles. "A treatment that can reduce bleeding with straightforward, once-weekly administration has the potential to fundamentally change how patients and caregivers approach this disease, offering control with a level of simplicity this community has long needed."

Hemophilia is typically diagnosed in early childhood and impacts more than 800,000 people worldwide.1 The inability of the blood to clot properly can increase the risk of painful bleeding, including inside the joints, which can cause joint scarring and damage.2,3 Children's joints have growing cartilage and bone, which makes them particularly susceptible to damage caused by repeated bleeding episodes.4

Inhibitors to factor replacement therapy limit treatment options for people living with hemophilia and are associated with an increased risk of uncontrolled bleeding.5 These inhibitory antibodies develop in approximately 20% of those with hemophilia A and 3% of those with hemophilia B.5 Many people living with inhibitors to FVIII and FIX are unable to continue taking factor replacement therapies as they no longer prevent or stop bleeding episodes, particularly in individuals who are refractory to immune tolerance induction therapy.