Niktimvo (axatilimab)
Niktimvo is approved for the treatment of chronic graft-versus host disease (cGVHD). Niktimvo contains the active ingredient axatilimab.
Niktimvo was approved for the treatment of chronic graft-versus-host disease (cGVHD), which can occur after a person receives a stem cell or bone marrow transplant from another person.
Immune cells, such as T cells, seek out and attack foreign objects, such as bacteria or viruses. Stem cell or bone marrow transplants (the ’graft’) contain immune cells from the donor. Graft-versus host disease occurs if the donor’s immune cells perceive healthy organs in the person receiving the transplant (the ‘host’) as foreign objects and attacks them.
Chronic GVHD is a long-lasting form of GVHD that can affect several organs. It often starts within the first year after transplant. It can have features of both an overactive immune response and a weakened immune system. This can lead to severe illness, reduced quality of life, and sometimes death. Symptoms can make everyday tasks difficult and may last for years.
Niktimvo was specifically approved for cGVHD, after the failure of at least 2 previous systemic (whole-body) therapies, in adults and children 6 years and older weighing at least 40 kg.
Niktimvo is a monoclonal antibody medicine. Antibodies are proteins produced by the immune system that can recognise and bind to specific targets in the body. Monoclonal antibodies are laboratory-produced versions of natural antibodies that all bind to the same target in the body.
Niktimvo antibodies are designed to block an activation ‘switch’ called colony stimulating factor-1 receptor (CSF1R). This switch helps certain immune cells, called macrophages, to develop and function. In cGVHD, these cells contribute to inflammation and play an important role in damaging tissues in different organs.
By blocking CSF1R, Niktimvo may lower the number of these cells and reduce their activity, which may help prevent the severity of chronic graft-versus-host disease.
The effectiveness and safety of Niktimvo were assessed in a clinical study where 241 patients were split into three dosage groups.
The main measure of benefit was the Overall Response Rate (ORR). The ORR is the percentage of patients who achieved a complete response (where all signs of cGVHD disappeared in every affected organ or site) or a partial response (a significant improvement in at least one organ or site). Responses to treatment were measured using well-established scoring systems for cGVHD.
Key findings from clinical studies
It was determined that the optimal dose of Niktimvo was 0.3 mg/kg given every 2 weeks. In this dose group:
- an ORR of 73.8% was achieved approximately 6 months after starting treatment.
- more than half the participants reported a clinically significant reduction in cGVHD symptoms at the 7-month mark.
- the length of the response was approximately 1.9 months.
- approximately 60% of responding patients remained alive and did not require a new treatment for at least 12 months following their initial response.
there were fewer and less severe adverse events (problems or side effects). Across this and other supporting studies, side effects included infusion-related reactions, infections, swelling around the eyes, and higher levels of enzymes that can indicate liver or muscle injury and pancreatitis. Most side effects were mild to moderate, expected, and clinically manageable.
The TGA decided that the application provided sufficient evidence supporting the safety and effectiveness of Niktimvo and the medicine can be registered in Australia.
More detailed information on why the TGA approved Niktimvo 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.
The Australian CMI for Niktimvo 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.
The Australian PI for Niktimvo can be accessed through the searchable TGA eBusiness Services or ARTG databases.
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