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Esperoct

Published
Product name
Esperoct
Active ingredient
Turoctocog alfa pegol
Submission type
New biological entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for

Esperoct (turoctocog alfa pegol) was approved for the following therapeutic use:

Esperoct, is a long-acting recombinant Factor VIII concentrate indicated for use in previously treated patients with haemophilia A for:

  • Routine prophylactic treatment to prevent or reduce the frequency of bleeding episodes
  • On-demand treatment and control of bleeding episodes
  • Peri-operative management of bleeding (surgical prophylaxis)

Esperoct does not contain von Willebrand factor, and therefore is not indicated in patients with von Willebrand’s disease.

How this medicine works

The factor VIII/von Willebrand factor complex consists of two molecules (factor VIII and von Willebrand factor) with different physiological functions. When infused into a patient with haemophilia, factor VIII binds to the patient’s von Willebrand factor. Activated factor VIII acts as a cofactor for activated factor IX, accelerating the conversion of factor X to activated factor X. Activated factor X converts prothrombin into thrombin. Thrombin then converts fibrinogen into fibrin and a clot can be formed.
Haemophilia A is a X-chromosomal hereditary disorder of blood coagulation due to decreased levels or absence of factor VIII:C that results in bleeding into joints, muscles or internal organs, either spontaneously or as a result of accidental or surgical trauma.
Turoctocog alfa pegol is a purified recombinant human factor VIII (rFVIII) product with 40 kDa polyethylene glycol (PEG), which is conjugated to protein. The PEG is attached to the O-linked glycan in the truncated B domain of rFVIII (turoctocog alfa). The mechanism of action of turoctocog alfa pegol is based on the substitution of inadequate or absent factor VIII in patients with haemophilia A.
When turoctocog alfa pegol is activated by thrombin at the injury site, the a3 region and the B domains containing the PEG are cleaved off, producing active recombinant factor VIII (rFVIIIa), which is similar in structure to native factor VIIIa.
This replacement therapy raises factor VIII plasma levels, temporarily correcting the factor VIII deficiency and bleeding tendency.
 

Why the TGA approved or did not approve this medicine

The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Esperoct was considered favourable for the therapeutic use approved.