Specialised Therapeutics has announced that NIKTIMVO (axatilimab) has been approved for use in Australia by the Therapeutic Goods Administration (TGA) for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and paediatric patients 6 years and older weighing at least 40 kg.
NIKTIMVO was assessed through the TGA’s Priority Review pathway and is the first approved anti-colony stimulating factor-1 receptor (anti-CSF-1R) antibody for cGVHD. Through its novel mechanism of action, NIKTIMVO specifically targets the key drivers of inflammation and fibrosis in tissues throughout the body, helping to address the serious and potentially life-threatening complications associated with cGVHD.
“Chronic GVHD is a significant long-term concern in patients with blood cancer requiring stem cell or bone marrow transplantation, and is characterised by multi-organ inflammation and fibrosis, leading to increased morbidity and non-relapse mortality,” said Jeff Szer, clinical haematologist at Peter MacCallum Cancer Centre and The Royal Melbourne Hospital.
“The Australian approval of NIKTIMVO is welcome news and will provide an important new treatment option for patients with chronic GVHD who continue to progress on other therapies, experience a higher symptom burden, and a decline in function and quality of life,” Szer said.
“I urge the Government and Specialised Therapeutics to determine an appropriate pathway to reimbursement to ensure eligible patients can have equitable access to this therapy as soon as possible.”
Graft-versus-host disease (GVHD) occurs when donated stem cells (the graft) mature into immune cells and attack the healthy cells in the patient’s body (the host). Approximately 600 allogeneic stem cell transplants — where a patient receives stem cells from a donor to replace their diseased blood cells and repair their bone marrow — are carried out in Australia each year. Chronic GVHD can occur at any time but typically develops 4-6 months after the transplant and affects approximately 40-50% of transplant recipients. In most cases, three or more organs are involved, ranging from mild skin rashes, to muscle weakness, pain and joint stiffness, which can impair overall functional capacity, to potentially life-threatening muti-organ failure.
In June 2025, Specialised Therapeutics entered into an exclusive partnering agreement with Incyte to commercialise NIKTIMVO in Australia, New Zealand and Singapore.
“Chronic GVHD remains an area of significant unmet need for Australians with blood cancer who have received donor stem cell or bone marrow transplants,” said Carlo Montagner, Specialised Therapeutics’ CEO.
“For patients who develop chronic GVHD, almost 50% will require at least three different therapies, reinforcing the need for additional effective treatment options.”
The development of NIKTIMVO was made possible due to important pre-clinical Australian research, which commenced in 2014. Scientists from QIMR Berghofer made the world-first discovery, identifying both the cellular process that causes cGVHD, and the antibody that could block this process and prevent the disease’s development.
“We are extremely proud to have secured local approval for NIKTIMVO, which would not have been possible without the pioneering work led by these Australian scientists over a decade ago,” Montagner said.
“Chronic GVHD may lead to debilitating symptoms that can be extremely difficult to treat. As the first country to obtain Marketing Authorisation for NIKTIMVO since it was approved by the US FDA in August 2024, we are pleased that it will soon be available for Australian prescribers and patients in need of new therapeutic options.”
The company is currently exploring the reimbursement of NIKTIMVO through the Pharmaceutical Benefits Scheme (PBS) for eligible adults and patients 6 years and older with cGVHD.
The TGA approval of NIKTIMVO was based on data from the pivotal phase 2 global AGAVE-201 clinical trial, which evaluated its safety and efficacy in 241 adult and some paediatric patients with refractory cGVHD who received at least two prior lines of systemic therapy. AGAVE-201 involved 121 clinical trial sites across 16 countries, including patients from Australia, Singapore, South Korea and Taiwan.
The overall response rate (ORR) among patients in the study who received NIKTIMVO was 74% at the approved dose (0.3 mg/kg every 2 weeks). Also, 60% of patients maintained a response at 12 months. In clinical studies, treatment with NIKTIMVO was generally well-tolerated, with a safety profile that was manageable and consistent with the mechanism of action of CSF-1R inhibition. The most common adverse events were dose-dependent transient laboratory abnormalities related to CSF-1R blockade.