ALTUVIIIO (efanesoctocog alfa)
ALTUVIIIO is approved for adults, adolescents and children with haemophilia A (congenital factor 8 (FVIII) deficiency). ALTUVIIIO contains the active ingredient efanesoctocog alfa.
ALTUVIIIO is an anti-haemophilic factor, also known as factor 8, used to treat adults, adolescents and children with haemophilia A (congenital factor 8 deficiency).
ALTUVIIIO is used in adults, adolescents and children with haemophilia A to:
- prevent and reduce the frequency of bleeding episodes
- treat and control bleeding episodes
- reduce bleeding before, during, and after surgery.
Haemophilia is an inherited bleeding disorder where the blood does not clot properly. This can lead to bleeding that is difficult to control. Haemophilia A is the most common type of haemophilia and is caused by a lack of clotting factor 8.
ALTUVIIIO does not contain von Willebrand factor. Therefore, it is not suitable for use in Von Willebrand's disease.
People with haemophilia A do not have enough factor 8 to control bleeding. Factor 8 is a protein essential for blood clotting.
ALTUVIIIO works by replacing the missing factor 8 in the blood allowing the blood to clot.
The effectiveness and safety of ALTUVIIIO were assessed in 2 main clinical studies:
- Study 1 assessed the effectiveness of ALTUVIIIO in adults and adolescents. A total of 159 patients aged 12 to 72 with severe haemophilia A were included in the study.
- Study 2 assessed the effectiveness of ALTUVIIIO in children under the age of 12. A total of 74 patients aged one to 11 with severe haemophilia A were included in the study.
The main goal of the studies was to determine the effectiveness of routine treatment with ALTUVIIIO to prevent and reduce bleeding episodes.
Other important measures included determining the effectiveness of on demand treatment to control bleeding episodes and to manage bleeding before, during and after surgery.
Key findings from clinical studies
In adult and adolescent patients:
- After 52 weeks of treatment with ALTUVIIIO patients had an average of 0.71 bleeding episodes per year.
- 78 patients had previous factor 8 treatment data available. In this group the average number of bleeding episodes per year was 0.69 with ALTUVIIIO and 2.96 with previous factor 8 treatment.
- A total of 362 bleeding episodes were treated. All but 1 bleeding episode was controlled with less than 2 injections of ALTUVIIIO.
- The most common side effects were joint pain and headache.
In children under the age of 12:
- Patients had an average of 0.6 bleeding episodes over 52 weeks of treatment with ALTUVIIIO.
- A total of 64 bleeding episodes were treated, 96.9% of these bleeding episodes were controlled with less than 2 injections of ALTUVIIIO.
- The most common side effects were fever and vomiting.
Data showed ALTUVIIIO was effective at managing bleeding before, during and after surgery with most patients requiring only one injection of ALTUVIIIO to prevent bleeding.
The TGA decided that the application provided sufficient evidence supporting the safety and effectiveness of ALTUVIIIO and the medicine can be registered in Australia.
More detailed information on why the TGA approved ALTUVIIIO will be published in the upcoming Australian Public Assessment Report (AusPAR).
For comprehensive information on potential side effects and risks, refer to the Consumer Medicine Information (CMI) leaflet or Product Information (PI) document.
This decision summary will not be updated to reflect any subsequent changes and may therefore not contain the most current information about the medicine. For the latest information, refer to the medicine’s CMI or PI.
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
Australian CMIs can be accessed through the searchable TGA eBusiness Services or ARTG databases.
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
Australian PIs can be accessed through the searchable TGA eBusiness Services or ARTG databases.
- ALTUVIIIO efanesoctocog alfa 1000 IU powder for injection vial (492505)
- ALTUVIIIO efanesoctocog alfa 2000 IU powder for injection vial (492506)
- ALTUVIIIO efanesoctocog alfa 250 IU powder for injection vial (492507)
- ALTUVIIIO efanesoctocog alfa 3000 IU powder for injection vial (492508)
- ALTUVIIIO efanesoctocog alfa 4000 IU powder for injection vial (492509)
- ALTUVIIIO efanesoctocog alfa 500 IU powder for injection vial (492510)
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