Specialised Therapeutics announced on May 3 that NIKTIMVO (axatilimab) has received approval from the Therapeutic Goods Administration for the treatment of chronic graft-versus-host disease (cGVHD) in Australia. The therapy is intended for adult and paediatric patients aged six years and older, weighing at least 40 kilograms, who have not responded to at least two prior lines of systemic therapy.
The approval marks the first time an anti-colony stimulating factor-1 receptor antibody has been authorised for cGVHD in Australia. This development is significant because cGVHD remains a serious complication following stem cell or bone marrow transplantation, leading to increased morbidity and non-relapse mortality among blood cancer patients. “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 Professor Jeff Szer, Clinical Haematologist at Peter MacCallum Cancer Centre and The Royal Melbourne Hospital.
Professor Szer also said, “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.” He called on authorities to establish reimbursement pathways so eligible patients can access the therapy promptly.
Graft-versus-host disease occurs when donated stem cells attack healthy cells in the recipient’s body. Each year about 600 allogeneic stem cell transplants are performed in Australia. Chronic GVHD typically develops four to six months after transplant but can occur at any time; it affects approximately 40-50% of recipients according to information cited by Specialised Therapeutics.
Carlo Montagner, Chief Executive Officer of Specialised Therapeutics, said: “Chronic GVHD remains an area of significant unmet need for Australians with blood cancer who have received donor stem cell or bone marrow transplants.” He added that nearly half these patients require three or more different therapies before achieving control over their condition.
Australian scientists contributed key pre-clinical research starting in 2014 that led to the development of NIKTIMVO. Montagner said: “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.” Australia is now the first country outside the United States where Marketing Authorisation has been granted since its US Food & Drug Administration approval in August 2024.
The TGA’s decision was based on results from AGAVE-201—a global phase two clinical trial involving 241 adults and some children—which showed an overall response rate of 74% at recommended dosing levels. Most common side effects included fatigue, elevated liver enzymes, infusion-related reactions as well as dose-dependent laboratory abnormalities associated with CSF-1R blockade.
Specialised Therapeutics plans further discussions regarding reimbursement through Australia’s Pharmaceutical Benefits Scheme (PBS). For more details about NIKTIMVO’s use or safety profile—including warnings related to infusion reactions—patients are advised to consult healthcare professionals or refer directly to official product information provided by ST Medical Information.
