| Type: | mab |
| Mab Type: | mab |
| Source: | zu |
| Target: | CSF-1R |
| Tradename: | Niktimvo |
| Dailymedid: | Axatilimab |
| Routes Of Administration: | Intravenous |
| Class: | Antineoplastic |
| Atc Prefix: | None |
| Legal Us: | Rx-only |
| Legal Us Comment: | [1] |
| Cas Number: | 2155851-88-8 |
| Drugbank: | DB16388 |
| Unii: | R96Z451BMC |
| Kegg: | D11952 |
| Synonyms: | axatilimab-csfr |
| C: | 6568 |
| H: | 10092 |
| N: | 1696 |
| O: | 2052 |
| S: | 48 |
Axatilimab, sold under the brand name Niktimvo, is a monoclonal antibody used for the treatment of chronic graft-versus-host disease. It is a blocker of the colony stimulating factor-1 receptor. It is given by injection into a vein.
Axatilimab was approved for medical use in the United States in August 2024.[2]
Axatilimab is indicated for the treatment of chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy in people weighing at least 40kg (90lb).
The most common adverse reactions include increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.
Efficacy was evaluated in AGAVE-201 (NCT04710576), a randomized, open-label, multicenter trial investigating three dosages of axatilimab in people with recurrent or refractory chronic graft-versus-host disease who had received at least two lines of systemic therapy and required additional treatment.
Axatilimab-csfr was granted orphan drug and fast track designations for the treatment of chronic graft-versus-host disease.
Axatilimab was approved for medical use in the United States in August 2024.[3]
Axatilimab is the international nonproprietary name.[4]