Prime Highlights-
- The FDA approved Pfizer’s Hympavzi for children with hemophilia B aged 6 to 11, marking the first non-factor therapy cleared for this age group.
- The approval also covers hemophilia A and B patients aged 12 and older with inhibitors, expanding the drug’s existing label.
Key Facts-
- Hympavzi is given via an auto-injector pen under the skin, replacing the need for frequent intravenous infusions.
- Late-stage trial data showed the drug significantly cut annual bleeding rates in adolescents and adults with inhibitors.
Background-
The U.S. Food and Drug Administration approved Pfizer’s drug Hympavzi to prevent and reduce bleeding episodes in children and certain patients living with hemophilia.
The approval makes Hympavzi the first non-factor therapy for pediatric patients with hemophilia B between the ages of 6 and 11, with or without inhibitors.
The regulator also widened the drug’s approved use to cover patients aged 12 and older with hemophilia A or B who have inhibitors, a type of antibody that blocks the production of blood-clotting proteins. Hympavzi already held approval for patients aged 12 and above without such inhibitors.
Doctors administer the drug under the skin through an auto-injector pen. This makes it a practical option over intravenous infusions, which patients typically need several times a week.
Hemophilia is a rare blood condition where the body lacks enough clotting proteins, leading to spontaneous or severe bleeding after injuries or surgery. Hemophilia A, the more common form, stems from low levels of clotting factor VIII. Hemophilia B occurs when the body does not produce enough clotting factor IX.
In a late-stage clinical trial, Hympavzi cut the mean treated annualized bleeding rate by a significant margin in adults and adolescents aged 12 and older with hemophilia A or B with inhibitors.
Pfizer said the drug offers a less burdensome treatment option for patients and their families. The latest approval adds to the drug’s growing label as Pfizer looks to expand its presence in the rare blood disorder space.
