Adzynma (apadamtase alfa/cinaxadamtase alfa)
Adzynma is approved for the treatment of ADAMTS13 deficiency in adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). Adzynma contains the active ingredient apadamtase alfa/cinaxadamtase alfa.
Adzynma has been approved to treat a very rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). People with cTTP are born without enough of the ADAMTS13 enzyme in their blood. ADAMTS13 normally prevents episodes in which thrombosis (harmful blood clots) can form and reduce blood flow to different organs, causing damage.
Adzynma is a laboratory-made version of the missing enzyme. At the time of approval, no satisfactory treatment was available for people with cTTP outside of a plasma infusion.
Adzynma is a treatment that replaces the missing ADAMTS13 enzyme in the blood. The ADAMTS13 enzyme processes a large protein called von Willebrand factor. This protein is involved in the first step of blood clotting at the site of injury. Von Willebrand factor helps cells called platelets stick together and attach to the walls of blood vessels, forming temporary clots to halt bleeding.
The ADAMTS13 enzyme cuts von Willebrand factor into smaller pieces to regulate its interaction with platelets. By breaking up von Willebrand factor, the enzyme prevents it from triggering the formation of harmful blood clots in normal circulation. It also helps to protect vital organs and keep blood platelet levels normal.
The active ingredient in Adzynma is a version of ADAMTS13 that is produced in a laboratory. This replaces the missing ADAMTS13 enzyme, preventing harmful blood clots from forming, and improving problems like bruising, bleeding, and anaemia.
The main evidence demonstrating that Adzynma is effective and safe for treating cTTP in people of all ages comes from 4 key clinical studies. The condition is extremely rare, resulting in only a small number of participants in the studies.
Key findings from clinical studies
- Adzynma was found to be more effective in reducing thrombocytopenia (low platelet count) episodes compared to the standard treatment.
- People treated with Adzynma, when compared to the standard treatment, had fewer symptoms of cTTP such as anaemia caused by damaged red blood cells, neurological problems and stomach pain.
- There were also fewer hospital visits, with similar quality of life, for participants treated with Adzynma, when compared to the standard treatments.
- Adzynma had similar treatment related side effects, and frequency, when compared to the standard treatment.
- Significantly, there were less transfusion reactions with Adzynma treatments when compared to the standard treatments. No participants developed antibodies that could block or weaken the effect of Adzynma.
The TGA decided that the application has provided sufficient evidence supporting the safety and effectiveness of Adzynma and the medicine can be registered for use in Australia.
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.
The Australian CMI for Adzynma can be accessed through the ARTG hyperlinks provided below.
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PI for Adzynma can be accessed through the ARTG hyperlinks provided below.
For health advice and information, including a symptom checker and service finder refer to the healthdirect website.
For advice on prescription medicines, over the counter medicines and other medicines (including complementary medicines) call Medicines Line.
For information on medicines subsidised by the Australian Government refer to the Pharmaceutical Benefits Scheme (PBS) website.
For data and reports on health and welfare topics in Australia refer to the Australian Institute of Health and Welfare website.