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<!-- Clinical data --> <!-- Clinical data -->
| pronounce = | pronounce =
| tradename = | tradename = Hympavzi
| Drugs.com = | Drugs.com = {{drugs.com|parent|Hympavzi}}
| MedlinePlus = | MedlinePlus =
| DailyMedID = <!-- DailyMed may use generic or brand name (generic name preferred) --> | DailyMedID = Marstacimab
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| pregnancy_AU_comment = | pregnancy_AU_comment =
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| class = | class = ] (TFPI)
| ATC_prefix = None | ATC_prefix = B02
| ATC_suffix = | ATC_suffix = BX11
| ATC_supplemental = | ATC_supplemental =
| biosimilars = | biosimilars =
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| legal_US_comment = <ref name="Hympavzi FDA label">{{cite web | title=Hympavzi- marstacimab-hncq injection, solution | website=DailyMed | date=5 November 2024 | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=a2cc631e-13a6-40c2-acf9-065ccedfb90a | access-date=11 November 2024}}</ref>
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| legal_UN_comment = | legal_UN_comment =
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| NIAID_ChemDB = | NIAID_ChemDB =
| PDB_ligand = | PDB_ligand =
| synonyms = PF-06741086 | synonyms = PF-06741086, marstacimab-hncq


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}} }}


'''Marstacimab''' is an experimental ] intended for the treatment of ] and B.<ref name="Hympavzi EPAR" /> It is being developed by Pfizer.<ref>{{cite press release | title=FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B | website=Pfizer | date=9 December 2023 | url=https://www.pfizer.com/news/press-release/press-release-detail/fda-and-ema-accept-marstacimab-regulatory-submissions | access-date=21 September 2024}}</ref> '''Marstacimab''', sold under the brand name '''Hympavzi''', is a ] medication used for the treatment of ] and ].<ref name="Hympavzi FDA label" /><ref name="FDA PR 20241011" /><ref name="Hympavzi EPAR" /> It is a tissue factor pathway inhibitor (TFPI) antagonist.<ref name="FDA PR 20241011" /> It was developed by Pfizer.<ref>{{cite press release | title=FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B | website=Pfizer | date=9 December 2023 | url=https://www.pfizer.com/news/press-release/press-release-detail/fda-and-ema-accept-marstacimab-regulatory-submissions | access-date=21 September 2024}}</ref> 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.<ref name="FDA PR 20241011" /> This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated.<ref name="FDA PR 20241011" /> This is expected to reduce or prevent the frequency of bleeding episodes.<ref name="FDA PR 20241011" />

The most common side effects include injection site reactions, headache, and itching (pruritis).<ref name="FDA PR 20241011" />

Marstacimab was approved for medical use in the United States in October 2024,<ref name="FDA PR 20241011" /> and in the European Union in November 2024.<ref name="Hympavzi PI">{{cite web | title=Hympavzi PI | website=Union Register of medicinal products | date=19 November 2024 | url=https://ec.europa.eu/health/documents/community-register/html/h1874.htm | access-date=21 November 2024}}</ref>

== Medical uses ==
Marstacimab is ] 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.<ref name="FDA PR 20241011" />

== Side effects ==
The US prescription label for marstacimab contains warnings and precautions about circulating blood clots (thromboembolic events), hypersensitivity, and embryofetal toxicity.<ref name="FDA PR 20241011" />

The most common side effects include injection site reactions, headache, and itching (pruritis).<ref name="FDA PR 20241011" />

== History ==
The US ] (FDA) approval of marstacimab is based on the BASIS study (NCT03938792),<ref name="Hympavzi FDA label" /> 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.<ref name="FDA PR 20241011" /> For the first six months of this study, participants received treatment with replacement factor either on-demand (33 participants) or prophylactically (83 participants).<ref name="FDA PR 20241011" /> These participants then received marstacimab prophylaxis for twelve months.<ref name="FDA PR 20241011" /> The primary measure of efficacy of marstacimab was the annualized bleeding rates of treated bleeds.<ref name="FDA PR 20241011" /> 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.<ref name="FDA PR 20241011" /> 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.<ref name="FDA PR 20241011" />

The FDA granted the application for marstacimab ] designation for this application.<ref name="FDA PR 20241011" /> The FDA granted the approval of Hympavzi to Pfizer Inc.<ref name="FDA PR 20241011" />


== Society and culture == == Society and culture ==
=== Legal status === === Legal status ===
In September 2024, the ] of the ] 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 {{convert|35|kg|lb}}, who have severe hemophilia A or B.<ref name="Hympavzi EPAR" /> The applicant for this medicinal product is Pfizer Europe Ma EEIG.<ref name="Hympavzi EPAR">{{cite web | title=Hympavzi EPAR | website=] (EMA) | date=19 September 2024 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/hympavzi | access-date=21 September 2024}} Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.</ref> In September 2024, the ] of the ] 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 {{convert|35|kg|lb}}, who have severe hemophilia A or B.<ref name="Hympavzi EPAR" /> The applicant for this medicinal product is Pfizer Europe Ma EEIG.<ref name="Hympavzi EPAR">{{cite web | title=Hympavzi EPAR | website=] (EMA) | date=19 September 2024 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/hympavzi | access-date=21 September 2024}} Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.</ref> Marstacimab was authorized for medical use in the European Union in November 2024.<ref name="Hympavzi PI" />

The US ] 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).<ref name="FDA PR 20241011">{{cite press release | title=FDA Approves New Treatment for Hemophilia A or B | website=U.S. Food and Drug Administration | date=1 October 2024 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-hemophilia-or-b | access-date=11 October 2024}} {{PD-notice}}</ref><ref>{{cite press release | title=U.S. FDA Approves Pfizer's Hympavzi (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors | publisher=Pfizer | via=Business Wire | date=11 October 2024 | url=https://www.businesswire.com/news/home/20241009505480/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-HYMPAVZI%E2%84%A2-marstacimab-hncq-for-the-Treatment-of-Adults-and-Adolescents-with-Hemophilia-A-or-B-Without-Inhibitors | access-date=12 October 2024}}</ref><ref>{{cite web | title=Novel Drug Approvals for 2024 | website=U.S. Food and Drug Administration | date=1 October 2024 | url=https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024 | access-date=8 November 2024}}</ref>


=== Names === === Names ===
Marstacimab is the ].<ref>{{cite journal | vauthors = ((World Health Organization)) | year = 2019 | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81 | journal = WHO Drug Information | volume = 33 | issue = 1 | hdl = 10665/330896 | hdl-access = free | author-link = World Health Organization }}</ref> Marstacimab is the ].<ref>{{cite journal | vauthors = ((World Health Organization)) | year = 2019 | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81 | journal = WHO Drug Information | volume = 33 | issue = 1 | hdl = 10665/330896 | hdl-access = free | author-link = World Health Organization }}</ref>

Marstacimab is sold under the brand name Hympavzi.<ref name="FDA PR 20241011" />


== References == == References ==

Latest revision as of 05:03, 23 December 2024

Medication

Pharmaceutical compound
Marstacimab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetTFPI
Clinical data
Trade namesHympavzi
Other namesPF-06741086, marstacimab-hncq
AHFS/Drugs.comHympavzi
License data
Routes of
administration		Intravenous
Drug classTissue factor pathway inhibitor (TFPI)
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC6304H9766N1678O2006S44
Molar mass142569.85 g·mol

Marstacimab, sold under the brand name Hympavzi, is a monoclonal antibody medication used for the treatment of hemophilia A and hemophilia B. It is a tissue factor pathway inhibitor (TFPI) antagonist. It was developed by Pfizer. 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. This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated. This is expected to reduce or prevent the frequency of bleeding episodes.

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

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

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.

Side effects

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

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

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. For the first six months of this study, participants received treatment with replacement factor either on-demand (33 participants) or prophylactically (83 participants). These participants then received marstacimab prophylaxis for twelve months. The primary measure of efficacy of marstacimab was the annualized bleeding rates of treated bleeds. 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. 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.

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

Society and culture

Legal status

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. The applicant for this medicinal product is Pfizer Europe Ma EEIG. Marstacimab was authorized for medical use in the European Union in November 2024.

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).

Names

Marstacimab is the international nonproprietary name.

Marstacimab is sold under the brand name Hympavzi.

References

  1. ^ "Hympavzi- marstacimab-hncq injection, solution". DailyMed. 5 November 2024. Retrieved 11 November 2024.
  2. ^ "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. ^ "Hympavzi PI". Union Register of medicinal products. 19 November 2024. Retrieved 21 November 2024.
  4. ^ "FDA Approves New Treatment for Hemophilia A or B". U.S. Food and Drug Administration (Press release). 1 October 2024. Retrieved 11 October 2024. Public Domain 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. "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.
  7. "Novel Drug Approvals for 2024". U.S. Food and Drug Administration. 1 October 2024. Retrieved 8 November 2024.
  8. World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl:10665/330896.

External links

  • Clinical trial number NCT03938792 for "Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B" at ClinicalTrials.gov
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