Marstacimab

Last updated

Marstacimab
Monoclonal antibody
Type Whole antibody
Source Human
Target TFPI
Clinical data
Trade names Hympavzi
Other namesPF-06741086, marstacimab-hncq
AHFS/Drugs.com Hympavzi
License data
Routes of
administration 		Intravenous
Drug class Tissue factor pathway inhibitor (TFPI)
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
Formula C6304H9766N1678O2006S44
Molar mass 142569.85 g·mol−1

Marstacimab, sold under the brand name Hympavzi, is a monoclonal antibody medication used for the treatment of hemophilia A and hemophilia B. [1] [4] [2] It is a tissue factor pathway inhibitor (TFPI) antagonist. [4] It was developed by Pfizer. [5] Marstacimab is a new type of medication that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor. [4] This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated. [4] This is expected to reduce or prevent the frequency of bleeding episodes. [4]

Contents

The most common side effects include injection site reactions, headache, and itching (pruritis). [4]

Marstacimab was approved for medical use in the United States in October 2024, [4] and in the European Union in November 2024. [3]

Medical uses

Marstacimab is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people aged twelve years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. [4]

Side effects

The US prescription label for marstacimab contains warnings and precautions about circulating blood clots (thromboembolic events), hypersensitivity, and embryofetal toxicity. [4]

The most common side effects include injection site reactions, headache, and itching (pruritis). [4]

History

The US Food and Drug Administration (FDA) approval of marstacimab is based on the BASIS study (NCT03938792), an open-label, multi-center study in 116 adult and pediatric male participants with either severe hemophilia A or severe hemophilia B, both without inhibitors. [4] For the first six months of this study, participants received treatment with replacement factor either on-demand (33 participants) or prophylactically (83 participants). [4] These participants then received marstacimab prophylaxis for twelve months. [4] The primary measure of efficacy of marstacimab was the annualized bleeding rates of treated bleeds. [4] In the participants receiving on-demand factor replacement during the first six months of the study, the estimated annualized bleeding rate was 38 compared to the estimated annualized bleeding rate during treatment with marstacimab of 3.2, showing that marstacimab was superior to on-demand factor replacement. [4] In the initial six-month period during which participants received prophylactic factor replacement, the estimated annualized bleeding rate was 7.85 and was 5.08 during the subsequent twelve months on marstacimab prophylaxis, showing that marstacimab provided similar bleeding rates. [4] [6]

Patients who completed the 12-month BASIS study were eligible to enroll in an open-label extension study (NCT05145127). [6]

The FDA granted the application for marstacimab orphan drug designation for this application. [4] The FDA granted the approval of Hympavzi to Pfizer Inc. [4]

Society and culture

In September 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Hympavzi, intended for prophylaxis of bleeding episodes in people aged twelve years of age and older, weighing at least 35 kilograms (77 lb), who have severe hemophilia A or B. [2] The applicant for this medicinal product is Pfizer Europe Ma EEIG. [2] Marstacimab was authorized for medical use in the European Union in November 2024. [3]

The US Food and Drug Administration approved marstacimab in October 2024, for people aged twelve years of age and older with hemophilia A and B without inhibitors (to prevent or reduce bleeding episodes). [4] [7] [8]

Names

Marstacimab is the international nonproprietary name. [9]

Marstacimab is sold under the brand name Hympavzi. [4]

Related Research Articles

<span class="mw-page-title-main">Haemophilia</span> Genetic disease involving blood clotting

Haemophilia, or hemophilia, is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or an altered level of consciousness.

<span class="mw-page-title-main">Haemophilia A</span> Medical condition

Haemophilia A is a blood clotting disorder caused by a genetic deficiency in clotting factor VIII, thereby resulting in significant susceptibility to bleeding, both internally and externally. This condition occurs almost exclusively in males born to carrier mothers due to X-linked recessive inheritance. Nevertheless, rare isolated cases do emerge from de novo (spontaneous) mutations.

Low-molecular-weight heparin (LMWH) is a class of anticoagulant medications. They are used in the prevention of blood clots and, in the treatment of venous thromboembolism, and the treatment of myocardial infarction.

<span class="mw-page-title-main">Dalteparin sodium</span> Pharmaceutical drug

Dalteparin is a low molecular weight heparin. It is marketed as Fragmin. Like other low molecular weight heparins, dalteparin is used for prophylaxis or treatment of deep vein thrombosis and pulmonary embolism to reduce the risk of a stroke or heart attack. Dalteparin acts by potentiating the activity of antithrombin III, inhibiting formation of both Factor Xa and thrombin. It is normally administered by self-injection.

<span class="mw-page-title-main">Tirofiban</span> Antiplatelet drug

Tirofiban, sold under the brand name Aggrastat, is an antiplatelet medication. It belongs to a class of antiplatelets named glycoprotein IIb/IIIa inhibitors. Tirofiban is a small molecule inhibitor of the protein-protein interaction between fibrinogen and the platelet integrin receptor GP IIb/IIIa and is the first drug candidate whose origins can be traced to a pharmacophore-based virtual screening lead.

Defibrotide, sold under the brand name Defitelio, is a mixture of single-stranded oligonucleotides that is purified from the intestinal mucosa of pigs. It is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant, with different limitations in the US and the European Union. It works by protecting the cells lining blood vessels in the liver and preventing blood clotting; the way it does this is not well understood.

Prothrombin complex concentrate (PCC), also known as factor IX complex, sold under the brand name Kcentra among others, is a combination medication made up of blood clotting factors II, IX, and X(3-factor PCC) or, when also containing factor VII as does Kcentra, 4-factor PCC. It is used to treat and prevent bleeding in hemophilia B if pure factor IX is not available. It may also be used for reversal of warfarin therapy. It is given by slow injection into a vein. Another product, activated prothrombin complex concentrate or FEIBA, may be used for acquired hemophilia.

<span class="mw-page-title-main">Tofacitinib</span> Medication

Tofacitinib, sold under the brand Xeljanz among others, is a medication used to treat rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, polyarticular course juvenile idiopathic arthritis, and ulcerative colitis. It is a janus kinase (JAK) inhibitor, discovered and developed by the National Institutes of Health and Pfizer.

<span class="mw-page-title-main">Apixaban</span> Anticoagulant medication

Apixaban, sold under the brand name Eliquis, is an anticoagulant medication used to treat and prevent blood clots and to prevent stroke in people with nonvalvular atrial fibrillation through directly inhibiting factor Xa. It is used as an alternative to warfarin to prevent blood clots following hip or knee replacement and in those with a history of prior clots. and does not require monitoring by blood tests or dietary restrictions. It is taken by mouth.

Recombinant factor VIIa (rfVIIa) is a form of blood factor VII that has been manufactured via recombinant technology. It is administered via an injection into a vein. It is used to treat bleeding episodes in people who have acquired haemophilia, among other indications. There are several disimilar forms, and biosimilars for each. All forms are activated.

<span class="mw-page-title-main">Cabotegravir</span> Medication for HIV/AIDS

Cabotegravir, sold under the brand name Vocabria among others, is a antiretroviral medication used for the treatment of HIV/AIDS. It is available in the form of tablets and as an intramuscular injection, as well as in an injectable combination with rilpivirine under the brand name Cabenuva.

Turoctocog alfa is a recombinant antihemophilic factor VIII used for the treatment of and prophylaxis of bleeding patients with haemophilia A. It is marketed by Novo Nordisk. It was approved in the United States, the European Union, and Japan in 2013.

<span class="mw-page-title-main">Sacituzumab govitecan</span> Antibody-drug conjugate

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Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. It was developed by BioMarin Pharmaceutical. Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with hemophilia A. It is an adeno-associated virus vector-based gene therapy. It is given by intravenous infusion.

<span class="mw-page-title-main">Nirmatrelvir/ritonavir</span> Antiviral combination medication

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References

  1. 1 2 "Hympavzi- marstacimab-hncq injection, solution". DailyMed. 5 November 2024. Retrieved 11 November 2024.
  2. 1 2 3 4 "Hympavzi EPAR". European Medicines Agency (EMA). 19 September 2024. Retrieved 21 September 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  3. 1 2 3 "Hympavzi PI". Union Register of medicinal products. 19 November 2024. Retrieved 21 November 2024.
  4. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 "FDA Approves New Treatment for Hemophilia A or B". U.S. Food and Drug Administration (Press release). 1 October 2024. Retrieved 11 October 2024.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  5. ↑ "FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B". Pfizer (Press release). 9 December 2023. Retrieved 21 September 2024.
  6. 1 2 "HYMPAVZI (marstacimab-hncq) injection, for subcutaneous use Initial U.S. Approval: 2024" (PDF). U.S. Food and Drug Administration. 1 October 2024. Retrieved 22 November 2024.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  7. ↑ "U.S. FDA Approves Pfizer's Hympavzi (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors" (Press release). Pfizer. 11 October 2024. Retrieved 12 October 2024– via Business Wire.
  8. ↑ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration. 1 October 2024. Retrieved 8 November 2024.
  9. ↑ World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl: 10665/330896 .


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