Niktimvo FDA Approval History
Last updated by Judith Stewart, BPharm on Aug 20, 2024.
FDA Approved: Yes (First approved August 14, 2024)
Brand name: Niktimvo
Generic name: axatilimab-csfr
Dosage form: Injection
Company: Incyte and Syndax Pharmaceuticals
Treatment for: Graft Versus Host Disease
Niktimvo (axatilimab-csfr) is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody used for the treatment of chronic graft-versus-host disease.
- Niktimvo is indicated 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 weighing at least 40 kg.
- Chronic graft-versus-host disease is a serious condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the organs of the transplant recipient. Chronic GVHD is characterized by the development of fibrotic tissue across multiple organ systems, including the skin and mucosa.
- Niktimvo contains axatilimab-csfr, a monoclonal antibody that binds to colony stimulating factor-1 receptors (CSF-1R) expressed on monocytes and macrophages. Niktimvo works to treat cGVHD by reducing the levels of circulating proinflammatory and profibrotic monocytes and monocyte-derived macrophages, and inhibits the activity of pathogenic macrophages in tissues.
- FDA approval of Niktimvo was based on data from the global AGAVE-201 study which demonstrated durable responses across all organs studied and patient subgroups. Among patients who received Niktimvo, 75% achieved an overall response rate within the first six months of treatment, with a median time to response of 1.5 months. Additionally, 60% maintained a response at 12 months.
- Niktimvo is administered as an intravenous infusion over 30 minutes, every 2 weeks.
- Warnings and precautions associated with Niktimvo include Infusion-related reactions and fetal harm.
- Common adverse reactions include infection (pathogen unspecified), viral infection, musculoskeletal pain, fatigue, nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.
Common laboratory abnormalities include increased AST, increased ALT, decreased phosphate, decreased hemoglobin, increased GGT, increased lipase, increased amylase, increased calcium, increased CPK, and increased ALP.
Development timeline for Niktimvo
Further information
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