Turoctocog alfa

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Turoctocog alfa
Clinical data
Trade names NovoEight, Esperoct
Other namesrecombinant antihemophilic factor
AHFS/Drugs.com Monograph
License data
Pregnancy
category
Routes of
administration 		Intravenous (IV)
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
ChEMBL
Chemical and physical data
Formula C7480H11379N1999O2194S68
Molar mass 166594.19 g·mol−1

Turoctocog alfa (trade name NovoEight) 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. [7] [9] [13] It was approved in the United States, the European Union, and Japan in 2013. [14] [15] [11] [9]

Contents

Medical uses

Turoctocog alfa is indicated for the treatment and prophylaxis of bleeding in adults and children with haemophilia A (congenital factor VIII deficiency). [11]

Turoctocog alfa pegol is indicated for the treatment and prophylaxis of bleeding in adults and children twelve years and above with haemophilia A (congenital factor VIII deficiency). [12]

In the safety and efficacy trial for prevention and treatment of bleeds, in hemophilia patients the success rate for treatment of bleeds was 84.5% (excluding bleeds for which there was no outcome reported) and out of a total of nine surgeries in nine patients performed during the trial, haemostasis was successful in all the surgeries and no treatment failures were reported. It is also used for perioperative management and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. Turoctocog alfa is not indicated for the treatment of von Willebrand disease. [16] [9]

Benefits and risks

In a study conducted with 150 patients aged twelve and above, adolescents after using turoctocog alfa as a treatment, had an average of 5.55 bleedings per year while the adults had an average of 6.68 bleeding per year. According to data, turoctocog alfa was considered an 'excellent' treatment for 403 out of 499 bleeding episodes. In another study involving 63 patients aged less than twelve years, children had an average of 5.33 bleedings per year after using turoctocog alfa as hemophilia treatment. In this study too turoctocog alfa was considered as an 'excellent' treatment for 116 out of 126 bleeding episodes. [17]

Alongside the benefits, a few of the common turoctocog alfa adverse effects would be injection site reaction, pyrexia and augmented liver enzyme levels. Rare cases of allergic reactions have been reported as well. There is a possibility of patients developing hypersensitivity to the drug since it contains traces of hamster proteins. Activity-neutralising antibodies may be developed whereby expected plasma factor VIII activity levels may not be achieved and thus the bleeding would not be controlled as needed. [16]

Preparation

As cell culture, Chinese hamster ovary cells (CHO) are used in order to acquire proper processing of factor VIII protein, that has demonstrated good efficacy in thrombin generation and clot formation in preclinical evaluations in murine (mouse) and canine (dog) models of hemophilia A and in patient-derived whole blood. [18]

Difference between NovoSeven and NovoEight

Even though both treatments are plasma-derived and recombinant analogues of blood clotting factors, NovoSeven is developed as a congenital FVIIa analogue for hemophilia A and B patients while NovoEight (turoctocog alfa) is for congenital FVIII deficiency. NovoSeven is called a bypassing agent because it skips the need for factor VIII or IX in people with inhibitors and activates factor X directly. [19]

Turoctocog alfa pegol

In February 2019, the FDA approved antihemophilic factor (recombinant), glycopegylated-exei (turoctocog alfa pegol) (Esperoct) for the treatment of hemophilia A. [20] [10] Turoctocog alfa pegol was approved for medical use in the European Union in June 2019. [12]

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 a decreased 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.

<span class="mw-page-title-main">Haemophilia B</span> Genetic X-linked recessive bleeding disorder

Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency.

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

Desmopressin, sold under the trade name DDAVP among others, is a medication used to treat diabetes insipidus, bedwetting, hemophilia A, von Willebrand disease, and high blood urea levels. In hemophilia A and von Willebrand disease, it should only be used for mild to moderate cases. It may be given in the nose, by injection into a vein, by mouth, or under the tongue.

<span class="mw-page-title-main">Coagulation factor VII</span> Mammalian protein found in humans

Coagulation factor VII is one of the proteins that causes blood to clot in the coagulation cascade, and in humans is coded for by the gene F7. It is an enzyme of the serine protease class. Once bound to tissue factor released from damaged tissues, it is converted to factor VIIa, which in turn activates factor IX and factor X.

Factor XIII deficiency occurs exceedingly rarely, causing a severe bleeding tendency. The incidence is one in a million to one in five million people, with higher incidence in areas with consanguineous marriage such as Iran that has the highest global incidence of the disorder. Most are due to mutations in the A subunit gene. This mutation is inherited in an autosomal recessive fashion.

<span class="mw-page-title-main">Factor VIII (medication)</span> Pharmaceutical drug

Factor VIII is a medication used to treat and prevent bleeding in people with hemophilia A and other causes of low factor VIII. Certain preparations may also be used in those with von Willebrand's disease. It is given by slow injection into a vein.

Moroctocog alfa is a recombinant antihemophilic factor genetically engineered from Chinese hamster ovary (CHO) cell line. Chemically it is a glycoprotein. It is manufactured by Genetics Institute, Inc. and used to control and prevent hemorrhagic bleeding and prophylaxis associated with surgery or to reduce the number of spontaneous bleeding episodes in patients with hemophilia A. It is partially a recombinant coagulation factor VIII since it has an amino acid sequence which compares to the 90 + 80 kDa form of factor VIII (BDDrFVIII). It also has posttranslational modifications which are similar to those of the plasma-derived molecule. It can not prevent hemorrhagic bleeding associated with von Willebrand's disease since it is not a von Willebrand factor.

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">Jeanne Lusher</span> American physician

Jeanne Marie Lusher, M.D. was an American physician, pediatric hematologist/oncologist, and a researcher in the field of bleeding disorders of childhood, and has served as the director of Hemostasis Program at the Children's Hospital of Michigan until her retirement on June 28, 2013.

Catridecacog, sold under the brand name Tretten in the US and NovoThirteen in the EU) is a class of recombinant factor XIII A-subunit based biopharmaceutical medicine, indicated in patients with a rare clotting disorder, congenital factor XIII A-subunit deficiency, which is a kind of Factor XIII deficiency. The medication prevents bleeding in patients with this condition, and has been approved by the U.S. Food and Drug Administration (FDA) for this use in the US in 2014. It was brought to market by Novo Nordisk.

Acquired haemophilia A (AHA) is a rare but potentially life-threatening bleeding disorder characterized by autoantibodies directed against coagulation factor VIII. These autoantibodies constitute the most common spontaneous inhibitor to any coagulation factor and may induce spontaneous bleeding in patients with no previous history of a bleeding disorder.

Susoctocog alfa, sold under the brand name Obizur, is a medication used for the treatment of bleeding episodes in adults with acquired haemophilia, a bleeding disorder caused by the spontaneous development of antibodies that inactivate factor VIII.

Vonicog alfa, sold under the brand names Vonvendi and Veyvondi, is a medication used to control bleeding in adults with von Willebrand disease. It is a recombinant von Willebrand factor.

Efmoroctocog alfa, sold under the brand name Elocta among others, is a medication for the treatment and prophylaxis of bleeding in people with hemophilia A. Efmoroctocog alfa is a recombinant human coagulation factor VIII, Fc fusion protein (rFVIIIFc). It is produced by recombinant DNA technology in a human embryonic kidney (HEK) cell line.

Damoctocog alfa pegol, sold under the brand name Jivi is a recombinant DNA-derived, Factor VIII concentrate medication used to treat hemophilia A.

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.

Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.

Efanesoctocog alfa, sold under the brand name Altuviiio, is a medication used for the treatment of hemophilia A.

Concizumab, sold under the brand name Alhemo, is a monoclonal antibody used for the treatment of hemophilia B. It is an anti-tissue factor pathway inhibitor.

References

  1. ↑ "Antihemophilic factor Use During Pregnancy". Drugs.com. 20 January 2020. Archived from the original on 16 July 2020. Retrieved 11 March 2020.
  2. ↑ "Prescription medicines: registration of new chemical entities in Australia, 2014". Therapeutic Goods Administration (TGA). 21 June 2022. Archived from the original on 10 April 2023. Retrieved 10 April 2023.
  3. ↑ "Turoctocog alfa (rch)" (PDF). Archived (PDF) from the original on 16 May 2022. Retrieved 29 April 2023.
  4. ↑ "Esperoct (Novo Nordisk Pharmaceuticals Pty Ltd)". Therapeutic Goods Administration (TGA). 23 June 2023. Retrieved 10 September 2023.
  5. ↑ "Esperoct". Therapeutic Goods Administration (TGA). 19 June 2023. Retrieved 10 September 2023.
  6. ↑ "Summary Basis of Decision (SBD) for Esperoct". Health Canada . 23 October 2014. Archived from the original on 31 May 2022. Retrieved 29 May 2022.
  7. 1 2 "NovoEight 250 IU powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 23 December 2019. Archived from the original on 17 January 2021. Retrieved 11 March 2020.
  8. ↑ "Esperoct 1000 IU powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 15 January 2021. Retrieved 2 October 2020.
  9. 1 2 3 4 "NovoEight (- antihemophilic factor, recombinant kit". DailyMed. 30 November 2018. Archived from the original on 24 March 2021. Retrieved 11 March 2020.
  10. 1 2 "Esperoct (antihemophilic factor- recombinant, glycopegylated-exei kit". DailyMed. 30 October 2019. Archived from the original on 26 March 2021. Retrieved 11 March 2020.
  11. 1 2 3 "NovoEight EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 30 October 2020. Retrieved 11 March 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  12. 1 2 3 "Esperoct EPAR". European Medicines Agency (EMA). 24 April 2019. Archived from the original on 24 October 2020. Retrieved 11 March 2020.
  13. ↑ Spreitzer H (7 January 2014). "Neue Wirkstoffe – NovoEight". Österreichische Apothekerzeitung (in German) (1/2014): 18.
  14. ↑ "Novo Nordisk annual report 2013" (PDF). Novo Nordisk. Archived (PDF) from the original on 20 November 2017. Retrieved 11 March 2020.
  15. ↑ "NovoEight". U.S. Food and Drug Administration (FDA). 19 July 2017. Archived from the original on 22 July 2017. Retrieved 11 March 2020.
  16. 1 2 "FDA Approval Document" (PDF). Food and Drug Administration . Archived (PDF) from the original on 7 February 2019. Retrieved 27 April 2014.
  17. ↑ "European Drug Assessment Report" (PDF). Archived (PDF) from the original on 2 May 2014. Retrieved 27 April 2014.
  18. ↑ Haddley K. "Turoctog alfa for the treatment of hemophilia A". Drugs today. Archived from the original on 2 May 2014. Retrieved 7 April 2014.
  19. ↑ "How NovoSeven Works". Archived from the original on 2 May 2014. Retrieved 27 April 2014.
  20. ↑ "Esperoct". U.S. Food and Drug Administration (FDA). 19 February 2019. STN: BL 125671. Archived from the original on 17 December 2019. Retrieved 11 March 2020.
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