Specialised Therapeutics (ST) has announced that Niktimvo has received approval from the Therapeutic Goods Administration (TGA) for the treatment of chronic Graft-Versus-Host Disease (cGVHD) in adult and paediatric patients aged six years and older, weighing at least 40 kg, who have failed at least two prior lines of systemic therapy.

The therapy was reviewed under the TGA’s Priority Review pathway and became the first approved anti-colony stimulating factor-1 receptor (CSF-1R) antibody for cGVHD. With its novel mechanism, Niktimvo targets key drivers of inflammation and fibrosis across multiple organs, addressing serious and potentially life-threatening complications associated with the disease.

Chronic GVHD is a common and severe complication following stem cell or bone marrow transplants, where donor immune cells attack the patient’s healthy tissues. It affects approximately 40–50 percent of transplant recipients and can involve multiple organs, leading to symptoms ranging from skin rashes and joint stiffness to organ failure. The condition remains a major unmet medical need, particularly for patients who do not respond to existing therapies.

The approval is supported by data from the global phase 2 AGAVE-201 clinical trial, which evaluated Niktimvo in 241 patients with refractory cGVHD. The study demonstrated a 74 percent overall response rate at the approved dose, with 60 percent of patients maintaining their response at 12 months. The treatment was generally well tolerated, with a safety profile consistent with its mechanism of action.

The development of Niktimvo is rooted in pioneering research conducted in Australia, where scientists first identified the cellular mechanisms driving cGVHD and the antibody capable of blocking disease progression. The therapy was initially approved in the United States in August 2024 and is now being introduced to the Australian market through a partnership between Specialised Therapeutics and Incyte.

Specialised Therapeutics is currently working toward securing reimbursement through Australia’s Pharmaceutical Benefits Scheme (PBS) to ensure broader patient access. The company emphasised that many cGVHD patients require multiple lines of therapy, highlighting the importance of introducing new and effective treatment options.

Ongoing studies are also exploring Niktimvo in combination therapies and additional indications, including idiopathic pulmonary fibrosis, as part of efforts to expand its clinical utility.