FDA approves new treatment for hemophilia A or B

The US Food and Drug Administration (FDA) on Friday approved US pharma giant Pfizer’s (NYSE: PFE) Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies).

“Today’s approval of Hympavzi provides patients with hemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process,” said Dr Ann Farrell, Director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “This new type of treatment underscores the FDA’s commitment to advance the development of innovative, safe and effective therapies,” she noted.

“Hympavzi is Pfizer’s second hemophilia treatment to receive FDA approval this year and is the latest meaningful scientific advancement in our more than 40-year commitment to improve care for people living with hemophilia,” said Aamir Malik, chief US commercial officer and executive vice president of Pfizer, adding: “We look forward to launching this latest medical breakthrough and to now offer three distinct classes of hemophilia medicines – an anti-TFPI, gene therapy, and recombinant factor treatments – that can meet the unique treatment needs of a wide range of patients.”