Pfizer's Hympavzi Wins USFDA Nod for Pediatric Hemophilia B, Broader Patient Use
Pfizer
New Delhi: The U.S. Food and Drug Administration (USFDA) has approved Pfizer's, opens new tab drug to prevent or reduce the frequency of bleeding episodes in children and in certain patients with hemophilia, the company said on Monday.
The nod makes Pfizer's Hympavzi the first non-factor therapy available for pediatric patients with hemophilia B aged 6 to 11 years with or without inhibitors.
The regulator also expanded the use to include those with hemophilia A or B aged 12 years and older with inhibitors, a type of antibody.
Hympavzi is already approved for patients aged 12 years and older without the antibodies that stop the production of the blood-clotting proteins.
It is given under the skin through an auto-injector pen, and is an alternative to intravenous infusions that are often administered multiple times a week.
Hemophilia is a rare condition in which the blood doesn't clot well because it doesn't have enough blood-clotting proteins, called clotting factors, causing spontaneous and severe bleeding following injuries or surgery.
Hemophilia A, the most common type, is caused by a lack of or low levels of the blood-clotting factor VIII. Meanwhile, Hemophilia B is caused by a deficiency of or low levels of blood-clotting factor IX.
In the late-stage trial, Hympavzi significantly reduced the mean treated annualized bleeding rate in adults and adolescents 12 years and older with hemophilia A or B with inhibitors.
(This story has been corrected to say that Hympavzi is the first non-factor therapy particularly for pediatric patients with hemophilia B, not all pediatric patients, in paragraph 2)
