Pfizer's new blockbuster drug Marstacimab wins marketing approval in China

On November 21, the NMPA official website showed that Pfizer's Class 1 new drug Marstacimab Injection has been approved for marketing in China.

The approval covers pediatric patients aged 12 years and older and adult patients weighing ≥35 kg who have severe Hemophilia A (congenital Factor VIII deficiency, FVIII <1%) without Factor VIII inhibitors, or severe Hemophilia B (congenital Factor IX deficiency, FIX <1%) without Factor IX inhibitors. These patients are indicated for routine prophylaxis to prevent bleeding episodes or reduce their frequency.

Marstacimab (PF-6741086) is a monoclonal antibody developed by Pfizer that targets the K2 domain of tissue factor pathway inhibitor (TFPI), representing a hemophilia rebalancing therapy. TFPI is a natural anticoagulant protein which functions to prevent thrombus formation and restore hemostasis.

The drug gained market approval in the United States, the European Union, and Japan in October, November, and December 2024, respectively. It is indicated for routine prophylaxis to reduce the frequency of bleeding episodes in patients 12 years of age or older weighing at least 35 kg, who have severe Hemophilia A (congenital Factor VIII deficiency, FVIII <1%) or severe Hemophilia B (congenital Factor IX deficiency, FIX <1%) without factor inhibitors.

With this approval, it has become the world's first once-weekly subcutaneous treatment for Hemophilia B, as well as the first fixed-dose regimen approved for both Hemophilia A and B.

Globally, Marstacimab has also received recognition from different regulatory agencies:

• In September 2019, the U.S. FDA granted Marstacimab Fast Track Designation.

• In October 2024, it was selected for the CDE's "Innovative Drug Designation" (also known as the "Care Program") pilot project, making it the inaugural participant in this initiative.