Source: Medicines Authority (MT) Revision Year: 2020 Publisher: Octapharma (IP) SPRL, Allée de la Recherche 65, 1070 Anderlecht, Belgium
Pharmacotherapeutic group: antihemorrhagics: blood coagulation factor IX.
ATC-code: B02BD04
Factor IX is a single chain glycoprotein with a molecular mass of about 68,000 Dalton. It is a vitamin-K dependent coagulation factor and it is synthesised in the liver. Factor IX is activated by factor XIa in the intrinsic coagulation pathway, and by the factor VII/tissue factor complex in the extrinsic pathway. Activated factor IX, in combination with activated factor VIII, activates factor X. Activated factor X converts prothrombin into thrombin.
Thrombin then converts fibrinogen into fibrin and a clot is formed.
Haemophilia B is a sex-linked hereditary disorder of blood coagulation due to decreased levels of factor IX and results in profuse bleeding into joints, muscles or internal organs, either spontaneously or as a result of accidental or surgical trauma. By replacement therapy the plasma levels of factor IX is increased, thereby enabling a temporary correction of the factor deficiency and correction of the bleeding tendencies.
A study in 25 children below 6 years of age was conducted. Thereof, 6 patients were previously untreated. The recovery after administration of >25 IU of OCTANINE/kg body weight was investigated during the first 3 months of treatment and after 12-24 months. The incremental recovery (geometric mean ± s.d., one-stage assay, actual potency) was calculated to be 0.8±1.4 and 0.9±1.3 %/IU/kg at the 1st and the 2nd assessment, respectively.
For OCTANINE the following results were achieved in one pharmacokinetic study with 13 Haemophilia B patients over 12 years of age (mean age 28 years, range 12-61 years):
| N=13 | Median | Mean | SD* | Minimum | Maximum |
|---|---|---|---|---|---|
| Incremental Recovery [IU/dl]/[IU/kg] | 1.2 | 1.3 | 0.5 | 0.8 | 2.4 |
| AUC*norm (IU x dl-1 x h x IU-1 x kg) | 32.4 | 37.7 | 13.0 | 24.5 | 64.0 |
| Half-life (h) | 27.8 | 29.1 | 5.2 | 22.0 | 36.8 |
| MRT* (h) | 39.4 | 40.0 | 7.3 | 30.2 | 51.6 |
| Clearance (ml x h-1 x kg) | 3.1 | 2.9 | 0.9 | 1.6 | 4.1 |
* AUC = area under the curve
* MRT = mean residence time
* SD = standard deviation
The incremental recovery was also tested in a second study. The meta-analysis of all recovery assessments (n=19) resulted in a mean recovery of 1.1 [IU/dl]/[IU/kg].
Human plasma coagulation factor IX (from the concentrate) is a normal constituent of the human plasma and acts like the endogenous factor IX.
Animal studies are limited and show no additional risks to those already mentioned in other sections of the SmPC.
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