Australia-based independent biopharmaceutical company Specialised Therapeutics (ST) announced on Sunday that its 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 40kg.

The company says that 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, it is claimed that 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.

In June 2025, ST entered into an exclusive partnering agreement with Incyte (NASDAQ:INCY) to commercialise NIKTIMVO in Australia, New Zealand and Singapore.

ST 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.