Singapore-based Specialised Therapeutics has announced that Australia’s Therapeutic Goods Administration (TGA) has approved Niktimvo (axatilimab) for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients aged 6 years and older weighing at least 40 kg. The axatilimab Australia approval makes the country the first outside the US to grant marketing authorization for the drug since the US FDA approved it in August 2024. Niktimvo is the first approved anti-colony stimulating factor-1 receptor (CSF-1R) antibody for cGVHD, and was assessed by the TGA under its Priority Review pathway.
The TGA indication mirrors the approved US label in its patient population and line-of-therapy requirement. The drug is administered intravenously at 0.3 mg/kg every two weeks. Specialised Therapeutics, which entered into an exclusive commercialization agreement with Incyte Corporation for Australia, New Zealand, and Singapore in June 2025, said it is currently pursuing reimbursement through Australia’s Pharmaceutical Benefits Scheme. Niktimvo is not yet listed on the PBS, meaning patient access will depend on the outcome of that reimbursement process. The drug carries a TGA Black Triangle designation, requiring reporting of suspected adverse events.
The TGA approval was based on data from AGAVE-201, a Phase II open-label, randomized, multicenter dose-ranging trial that enrolled 241 adult and pediatric patients with recurrent or refractory active cGVHD whose disease had progressed after at least two prior therapies. The trial’s primary endpoint was the proportion of patients achieving an objective response by cycle 7 day 1, as defined by 2014 NIH Consensus Criteria. Among patients receiving the approved dose of 0.3 mg/kg every two weeks, the overall response rate was 74% (95% CI: 63, 83), and 60% of patients maintained a response at 12 months. The most common adverse reactions, occurring in 15% or more of patients, were fatigue, elevated liver enzymes, and infusion-related reactions, with grade 3 or 4 infusion-related reactions in 1.3% of patients.