Pfizer Submits NDA in China for Marstacimab, a Once-Weekly Subcutaneous Therapy for Hemophilia A and B

Aug 13, 2024 08:09 CST Updated 08:09

Pfizer

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Today (August 13), the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) announced that Pfizer, Inc. has submitted an application.Class 1Therapeutic Biological ProductsMatasumab InjectionSubmitted for marketing approval in China and accepted。Matasumab (marstacimab ，PF-06741086）It was developed by Pfizer.New Hemophilia Therapy Requires Only Once-Weekly Subcutaneous Injection.

It is worth mentioning that Marstacimab Injection treats hemophilia by targeting tissue factor pathway inhibitor (TFPI).Its mechanism of action is different from that of factor replacement drugs.Expected efficacy is not affected by the presence of inhibitors., is expected to change the major pain point of inhibitor production in clinical treatment. Moreover, itsOnce-weekly subcutaneous injection offers greater convenience and is expected to improve compliance.。

Screenshot source: CDE official website

Hemophilia is a hereditary bleeding disorder caused by genetic defects. Among them, Hemophilia A is caused by the lack of coagulation factor VIII.HemophiliaB is caused by coagulation factor IX. Patients experience prolonged bleeding after injury or surgery, and spontaneous bleeding may even occur in muscles, joints, and organs, which can be life-threatening in severe cases.

Currently,TargetingHemophiliaThe main treatment method isCoagulation factor replacement therapy, but many patients will develop coagulation factor inhibitors (antibodies produced by the immune system) during treatment, which in turn affects the treatment efficacy. Based on this, the industry is developing various new types.Therapy, to solve this problem. Among them, Tissue Factor Pathway Inhibitor (TFPI) targeted therapy is one of the therapies under research. TFPI isA naturally occurring anticoagulant protein in the human body。

In May 2023, Pfizer announced a key Phase 3 clinical trial of BASIS.The trial met the primary endpoint.Data show that, compared with preventive and on-demand intravenous treatment regimens,Matasimab InjectionThe reduction in annualized bleeding rate (ABR) was statistically significant and clinically relevant.

Compared with the patient cohort receiving on-demand factor replacement intravenous therapy during the导入期,Matasumab InjectionDemonstrated superiority with a 92% reduction in bleeding rate.
Compared with the cohort receiving preventive treatment,Matasumab InjectionIt also has superiority, with an annualized bleeding rate reduced by 35%.

Public information shows,Pfizer is also conducting anotherMatasimabPhase 3 trial,Study its use in treating patients with hemophilia A and B, with or without inhibitors of coagulation factors.Previously, Matasumab hadGranted Fast Track and Orphan Drug designations by the U.S. FDA.Currently, the FDA alsoCorrectUnder ReviewMatacizumabMarketing Authorization Application.

August 2020,Matacizumab Approved for Clinical Use in China for the First Time, Indication is:For routine prophylaxis treatment in patients with hemophilia A or hemophilia B, aged ≥12 years, with or without inhibitors, to prevent or reduce the frequency of bleeding episodes.

According toAccording to the information from the China Drug Clinical Trial Registration and Information Disclosure Platform, currently aMarstacimab in Pediatric Hemophilia Subjects With or Without InhibitorsOpen-label Phase 3 Study of Preventive TreatmentIn progress.

Screenshot Source: Drug Clinical Trial Registration and Information Disclosure Platform

The studyThe purpose is to evaluate in pediatric subjects aged ≥1 to <18 years with severe Hemophilia A or moderate-to-severe to severe Hemophilia B (defined as FVIII activity <1% or FIX activity ≤2%, respectively) with or without inhibitors.marstacimabThe safety, tolerability, and efficacy of preventive treatment.About100 pediatric subjects will be enrolled in thisResearch, and accept1Year'sTreatment.

This timeMarstacimab submitted for marketing approval in China and was accepted by the CDE, indicating that this innovative hemophilia therapy is expected to be approved for marketing in China in the near future, providing patients with more treatment options.

References:

[1] Official website of the Center for Drug Evaluation, National Medical Products Administration of China. Retrieved Aug 13, 2024, from https://www.cde.org.cn/main/xxgk/listpage/da6efd086c099b7fc949121166f0130c

[2] Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial. Retrieved May 30, 2023 from https://www.pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-marstacimab-results-pivotal-phase

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