Pfizer's TFPI-Targeting Hemophilia Therapy Marstacimab Receives First Clinical Approval in China

Source | Medical Horizon

Today, the website of the Center for Drug Evaluation (CDE) under China’s National Medical Products Administration disclosed that Pfizer’s marstacimab (PF-06741086) injection has received implicit clinical trial approval in China. The indicated indication is “for routine prophylactic treatment to prevent bleeding episodes or reduce the frequency of bleeding episodes in patients aged ≥12 years with hemophilia A or hemophilia B, with or without inhibitors.” Marstacimab is an investigational innovative therapy targeting tissue factor pathway inhibitor (TFPI) and has been granted Fast Track designation and Orphan Drug designation in the United States. This marks the first clinical trial approval for this drug in China.

Image source: CDE screenshot

Currently, clotting factor replacement therapy is the primary treatment for patients with hemophilia. However, many patients develop inhibitors (antibodies produced by the immune system) against clotting factors during treatment, thereby compromising therapeutic efficacy. Consequently, researchers are developing various other investigational therapies to provide more treatment options for hemophilia patients who have developed inhibitors.

Tissue Factor Pathway Inhibitor (TFPI)-Targeted Therapy is One of the Investigational Therapies Poised to Address the Unmet Needs of This Patient Population. TFPI is a naturally occurring anticoagulant protein in the human body that inhibits the alternative pathway of blood coagulation at its early stage. Investigational therapies targeting TFPI can be used to treat patients with hemophilia A and B, regardless of inhibitor status.

Marstacimab is an investigational TFPI-targeted therapy developed by Pfizer, intended for the treatment of hemophilia. In September 2019, the drug was granted Fast Track designation by the U.S. FDA for the treatment of hemophilia. Additionally, marstacimab has received orphan drug designation in both the United States and Europe.

Currently, marstacimab has entered Phase 3 clinical trials globally and has not yet been approved for market launch. Information from the ClinicalTrials.gov website indicates that Pfizer has completed a Phase 2 clinical trial in patients with hemophilia, aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of marstacimab in patients with severe hemophilia A or hemophilia B.

In addition, Pfizer is conducting two clinical studies of marstacimab in patients with hemophilia: one is an open-label Phase 3 clinical trial designed to compare the efficacy of marstacimab versus standard care in adolescents and adults with severe hemophilia A or B, with or without inhibitors; the other is a Phase 2 clinical trial aimed at evaluating the safety, tolerability, and efficacy of long-term treatment with marstacimab in patients with severe hemophilia.

Hemophilia is a monogenic X-linked recessive disorder, primarily characterized by impaired generation of active thromboplastin. Hemophilia A is caused by a deficiency in coagulation factor VIII, while hemophilia B is caused by a deficiency in coagulation factor IX. Patients with hemophilia may experience prolonged bleeding times after injury or surgery compared to the general population. Additionally, they may suffer from spontaneous bleeding into muscles, joints, and organs, which can be life-threatening in severe cases.

References

[1] Center for Drug Evaluation, National Medical Products Administration of China. Retrieved Aug 03, 2020, from http://www.cde.org.cn/news.do?method=changePage&pageName=service&frameStr=25

[2] Pfizer rare disease pipeline. From https://www.pfizer.com/science/rare-diseases/pipeline

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