The FDA has approved Niktimvo for treating chronic graft-versus-host disease (GVHD) in adults and children weighing over 40 kg. Chronic GVHD occurs after an allogeneic stem cell transplant when the donor cells attack the recipient’s organs.
Approximately 42% of transplant recipients develop chronic GVHD, with nearly 50% requiring three or more treatment lines. Niktimvo, an anti-CSF-1R antibody, targets a novel mechanism of action to address the complications associated with chronic GVHD.
The FDA approval is based on data from the AGAVE-201 study, which included 241 patients with refractory chronic GVHD who had received prior systemic therapy. Niktimvo at 0.3 mg/kg every two weeks resulted in a 75% overall response rate within six months and a 60% response rate maintained at 12 months.
Additionally, the study showed improvements in symptoms, with 56% of patients achieving a ≥7-point improvement in the modified Lee Symptom Scale score. Niktimvo demonstrated complete and partial responses in all organs affected by chronic GVHD, including the lower and upper gastrointestinal tracts, skin, and liver.
