FDA Approves Incyte And Syndaxs’ Niktimvo For Chronic Graft-Versus-Host Disease (GVHD)
Niktimvo gets FDA approval for chronic GVHD, offering hope to patients after two prior therapies.
Breaking News
Jan 16, 2025
Mrudula Kulkarni

Incyte and Syndax Pharmaceuticals have announced that the FDA has approved their drug, Niktimvo™ (axatilimab-csfr), for the treatment of chronic graft-versus-host disease (GVHD) in patients who haven't responded to at least two prior therapies. The drug will be available in 9 mg and 22 mg vial sizes and should be ready for order in the U.S. by early February. Niktimvo is the first treatment of its kind that targets a specific receptor (CSF-1R) to help reduce inflammation and scarring in patients with chronic GVHD.
Hervé Hoppenot, Chief Executive Officer, Incyte, said in a statement, "We are thrilled to build on our strong commitment to the GVHD community with the U.S. launch of Niktimvo, a first-in-class therapeutic agent that has demonstrated remarkable responses in patients with chronic GVHD whose response was suboptimal after at least two prior lines of systemic therapy. Our deep understanding of chronic GVHD and our connections in the clinical community will support a successful launch, in partnership with Syndax, of this important medicine for patients."
This approval is based on positive results from a large trial (AGAVE-201), which showed that 75% of patients on the drug experienced improvement after six months. However, like all medications, Niktimvo comes with potential risks. Around 44% of patients experienced serious side effects, including infections and respiratory problems. Some patients had to stop or reduce their dose because of these reactions. For treatment, the approved dose of Niktimvo is 0.3 mg per kg of body weight, administered as an intravenous infusion every two weeks.
"As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a breakthrough for patient care. Together with Incyte, we look forward to executing a robust commercial launch and advancing the treatment paradigm for patients with chronic GVHD who have progressed after at least two lines of systemic therapy," said Michael Metzger, Chief Executive Officer, Syndax.
The drug will be distributed through speciality suppliers to ensure it’s easy for doctors and patients. Incyte and Syndax are working to make Niktimvo accessible to as many patients as possible, with support programs available to help with financial assistance and education. In addition, the drug has been added to treatment guidelines for chronic GVHD, which means doctors now have clear recommendations for when to use it. Incyte and Syndax jointly promote Niktimvo in the U.S., while Incyte has exclusive rights to sell it outside the U.S.