The approval of HYMPAVZI is based on the results of a phase III trial study, which showed that compared with factor based prophylaxis and on-demand treatment, the drug can significantly reduce bleeding rates in eligible hemophilia A and hemophilia B patients without inhibitors.

HYMPAVZI is the first weekly subcutaneous injection regimen provided in the United States for eligible hemophilia B patients. At the same time, it is also the first regular treatment provided to eligible hemophilia A and hemophilia B patients through pre filled injection pens or syringes.

Recently, Pfizer's innovative drug HYMPAVZI ™ Mataximab hncq has been approved by the US Food and Drug Administration (FDA) for the regular treatment of hemophilia A (congenital factor VIII deficiency) or hemophilia B (congenital factor IX deficiency) patients over 12 years old without coagulation factor inhibitors, in order to reduce bleeding episodes.

HYMPAVZI is the first and only approved anti tissue factor pathway inhibitor (anti TFPI) for the treatment of hemophilia A and hemophilia B in the United States. It is also the first innovative hemophilia therapy in the United States to be administered via a pre filled automatic injection pen, requiring only weekly subcutaneous injection and convenient administration.

The approval of HYMPAVZI is a significant advancement in reducing bleeding for hemophilia A and hemophilia B patients without inhibitors. Its safety is controllable, and the treatment method of subcutaneous administration once a week is very simple, "said Suchitra S. Acharya, Director of the Hemostasis and Thrombosis Center at Northwell Medical Center and Head of the Hemorrhagic Disease and Thrombosis Program at Cohen Children's Medical Center." HYMPAVZI aims to meet the important needs of hemophilia patients and reduce their treatment burden, especially for many patients who require frequent and time-consuming intravenous infusion therapy

Hemophilia is a rare genetic blood disorder caused by a deficiency in coagulation factors (hemophilia A lacks FVIII, hemophilia B lacks FIX), affecting over 800000 people worldwide. Hemophilia is usually diagnosed in early childhood, which can cause coagulation dysfunction, increase the risk of recurrent intra-articular bleeding, and ultimately lead to permanent joint damage. Although significant progress has been made in the treatment of hemophilia in recent years, many hemophilia patients still experience bleeding episodes and require frequent intravenous infusions multiple times a week to control the condition. four

HYMPAVZI is Pfizer's second hemophilia treatment drug approved by the FDA this year, and it is also the latest significant scientific progress we have made in over 40 years to improve the treatment of hemophilia patients, "said Aamir Malik, Pfizer's Chief Business Officer and Executive Vice President in the United States. We look forward to launching this latest medical breakthrough as soon as possible, in order to provide innovative treatment drugs for three types of hemophilia simultaneously - anti TFPI, gene therapy, and recombinant factor therapy - to meet the personalized treatment needs of patients

The results of HYMPAVZI's Phase III BASIS trial (NCT03938792) support the approval in the United States. This study shows that compared to factor prevention (RP) and on-demand treatment (OD), HYMPAVZI can reduce the annualized bleeding rate (ABR) of hemophilia A and hemophilia B patients without inhibitors by 35% and 92%, respectively, after 12 months of active treatment. The safety of HYMPAVZI is consistent with the results of Phase I/II studies. The most commonly reported adverse reactions in research (≥ 3% of patients) are injection site reactions, headaches, and itching.

The hemophilia patient population is constantly seeking advanced treatment methods that can improve their quality of life, "said Phil Gattone, President and CEO of the National Hemorrhagic Disease Foundation in the United States

In August of this year, the Drug Evaluation Center of the National Medical Products Administration accepted the market application for Mataximab, an innovative drug that is expected to bring a new pattern to the treatment of hemophilia in China and provide patients with more treatment options. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has also given a positive opinion on the use of mataximab for the regular treatment of severe hemophilia A and hemophilia B patients aged 12 years and above without inhibitors to reduce bleeding episodes. In addition to HYMPAVZI, Pfizer's hemophilia B gene therapy BEQVEZ ™ Fidanacogene elaparvovec has recently received regulatory approval from the United States, European Union, and Canada, and Pfizer has also announced positive results from the Phase 3 clinical trial of Girocogene fitelparvovec for hemophilia A.