Axatilimab

Axatilimab
Monoclonal antibody
Type	Whole antibody
Source	Humanized
Target	CSF-1R
Clinical data
Trade names	Niktimvo
Other names	axatilimab-csfr
AHFS/Drugs.com	Niktimvo
License data	US DailyMed: Axatilimab
Routes of administration	Intravenous
Drug class	Antineoplastic
ATC code	None
Legal status
Legal status	US: ℞-only^[1]
Identifiers
CAS Number	2155851-88-8
DrugBank	DB16388
UNII	R96Z451BMC
KEGG	D11952
Chemical and physical data
Formula	C₆₅₆₈H₁₀₀₉₂N₁₆₉₆O₂₀₅₂S₄₈
Molar mass	147185.68 g·mol⁻¹

Axatilimab, sold under the brand name Niktimvo, is a monoclonal antibody used for the treatment of chronic graft-versus-host disease.^[1] It is a blocker of the colony stimulating factor-1 receptor.^[1] It is given by injection into a vein.^[1]

Axatilimab was approved for medical use in the United States in August 2024.^[1]^[2]

Medical use

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 40 kilograms (88 lb).^[1]^[2]

Adverse effects

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.^[2]

History

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.^[2]

Axatilimab-csfr was granted orphan drug and fast track designations for the treatment of chronic graft-versus-host disease.^[2]

Society and culture

Legal status

Axatilimab was approved for medical use in the United States in August 2024.^[2]^[3]

Names

Axatilimab is the international nonproprietary name.^[4]

References

^ ^a ^b ^c ^d ^e ^f "Niktimvo- axatilimab-csfr injection". DailyMed. 16 August 2024. Retrieved 5 September 2024.
^ ^a ^b ^c ^d ^e ^f "FDA approves axatilimab-csfr for chronic graft-versus-host disease". U.S. Food and Drug Administration. 14 August 2024. Archived from the original on 15 August 2024. Retrieved 15 August 2024. This article incorporates text from this source, which is in the public domain.
^ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 1 October 2024. Retrieved 29 November 2024.
^ World Health Organization (2020). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83". WHO Drug Information. 34 (1). hdl:10665/339768.

External links

Clinical trial number NCT04710576 for "A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD) (AGAVE-201)" at ClinicalTrials.gov

Immunosuppressive drugs / Immunosuppressants (L04)

Intracellular
(initiation)

Antimetabolites	purine synthesis inhibitors Azathioprine Mycophenolic acid dihydroorotate dehydrogenase inhibitors Leflunomide Teriflunomide antifolate Methotrexate
Macrolides/ other IL-2 inhibitors	FKBP/Cyclophilin/Calcineurin Ciclosporin Pimecrolimus Tacrolimus Voclosporin Abetimus Gusperimus
IMiDs	Lenalidomide Pomalidomide Thalidomide PDE4 inhibitor Apremilast

Intracellular
(reception)

IL-1 receptor antagonists	Anakinra
mTOR	Everolimus Ridaforolimus Sirolimus Temsirolimus Umirolimus Zotarolimus

Extracellular

Antibodies

Monoclonal

Serum target (noncellular)	Complement component 5 Eculizumab TNF Adalimumab Afelimomab Certolizumab pegol Golimumab Infliximab Nerelimomab Interleukin Anakinra Basiliximab Bimekizumab Briakinumab Brodalumab Canakinumab Daclizumab Guselkumab Ixekizumab Netakimab Olokizumab Rilonacept Risankizumab Sarilumab Satralizumab Secukinumab Siltuximab Sirukumab Spesolimab Tildrakizumab Tocilizumab Ustekinumab Interleukin 5 Mepolizumab Immunoglobulin E Omalizumab Interferon Faralimomab IL-6 Elsilimomab IL-12, IL-13, and IL-23 Lebrikizumab Ustekinumab IL-17A Secukinumab
Cellular target	CD3 Muromonab-CD3 Otelixizumab Teplizumab Visilizumab CD4 Clenoliximab Keliximab Zanolimumab CD11a Efalizumab CD18 Erlizumab CD20 Obinutuzumab Ocrelizumab (+hyaluronidase) Pascolizumab Rituximab Ublituximab CD23 Gomiliximab Lumiliximab CD40 Teneliximab Toralizumab CD62L/L-selectin Aselizumab CD80 Galiximab CD147/Basigin Gavilimomab CD154 Frexalimab Ruplizumab BLyS Belimumab CAT Bertilimumab Lerdelimumab Metelimumab Integrin Natalizumab Vedolizumab Interleukin-6 receptor Tocilizumab LFA-1 Odulimomab IL-2 receptor/CD25 Basiliximab Daclizumab Inolimomab T-lymphocyte (Zolimomab aritox)
Unsorted	Alemtuzumab Anifrolumab Atorolimumab Begelomab Cedelizumab Emapalumab Fontolizumab Inebilizumab Maslimomab Morolimumab Ofatumumab Pexelizumab Reslizumab Rovelizumab Siplizumab Talizumab Telimomab aritox Teprotumumab Vapaliximab Vepalimomab

Polyclonal

-cept (Fusion)

CTLA-4
- Abatacept
- Belatacept
TNF inhibitor
- Etanercept
- Opinercept
Aflibercept
Alefacept
Rilonacept

Unsorted

Monoclonal antibodies for tumors

Tumor

Human	Adecatumumab^§ Amivantamab Ascrinvacumab^§ Atezolizumab (+hyaluronidase) Balstilimab^† Botensilimab Cixutumumab^§ Conatumumab^§ Cosibelimab Daratumumab Drozitumab^§ Duligotumab^§ Dusigitumab^§ Enfortumab vedotin Enoticumab^§ Figitumumab^§ Flanvotumab^§ Ganitumab^† Glembatumumab vedotin^† Intetumumab^§ Ipilimumab Iratumumab^§ Istiratumab Icrucumab^§ Lexatumumab^§ Lucatumumab^§ Mapatumumab^§ Narnatumab^§ Necitumumab Nesvacumab^§ Nivolumab^# Ofatumumab Olaratumab^† Panitumumab Patritumab^§ Pembrolizumab^# Pritumumab^§ Radretumab^§ Ramucirumab Rilotumumab^† Robatumumab^§ Seribantumab^§ Sugemalimab Tarextumab^§ Tisotumab vedotin Teprotumumab^† Tovetumab^§ Vantictumab^§ Votumumab^§ Zalutumumab^†
Mouse	Abagovomab Altumomab pentetate Anatumomab mafenatox Arcitumomab Bectumomab Blinatumomab Capromab pendetide Detumomab Edrecolomab Ibritumomab tiuxetan Igovomab Lilotomab Minretumomab Mitumomab Nacolomab tafenatox Moxetumomab pasudotox Naptumomab estafenatox Oregovomab Pemtumomab^† Racotumomab Satumomab pendetide Solitomab Taplitumomab paptox Nofetumomab merpentan Pintumomab Tenatumomab Tositumomab
Chimeric	Amatuximab^§ Bavituximab^§ Brentuximab vedotin Carotuximab^† Cetuximab Derlotuximab biotin^§ Dinutuximab Ecromeximab^§ Ensituximab^§ Futuximab^§ Girentuximab^† Indatuximab ravtansine^§ Isatuximab Loncastuximab tesirine Margetuximab Mirvetuximab soravtansine Rituximab^# Siltuximab Ublituximab Zolbetuximab
Humanized	Abituzumab^§ Alemtuzumab Axatilimab Belantamab mafodotin Bevacizumab Bivatuzumab mertansine^§ Brontictuzumab^§ Cantuzumab mertansine^§ Cantuzumab ravtansine^§ Cirmtuzumab Citatuzumab bogatox^§ Clivatuzumab tetraxetan Cofetuzumab pelidotin Dacetuzumab^§ Demcizumab^§ Dalotuzumab^§ Denintuzumab mafodotin^§ Elotuzumab Emactuzumab Emibetuzumab^§ Enoblituzumab^§ Epcoritamab Etaracizumab^§ Farletuzumab^§ Ficlatuzumab^§ Flotetuzumab^§ Gemtuzumab ozogamicin Glofitamab Imgatuzumab^§ Inotuzumab ozogamicin Labetuzumab^§ Lifastuzumab vedotin^§ Lintuzumab^§ Lorvotuzumab mertansine^§ Lumretuzumab^§ Matuzumab^§ Milatuzumab^§ Naxitamab Nimotuzumab^† Obinutuzumab Ocaratuzumab^§ Odronextamab Otlertuzumab^§ Onartuzumab^§ Oportuzumab monatox^† Parsatuzumab^§ Pertuzumab Pinatuzumab vedotin^§ Polatuzumab vedotin Rosmantuzumab Rovalpituzumab tesirine^† Sacituzumab govitecan Sibrotuzumab^§ Simtuzumab^§ Sofituzumab vedotin^§ Tacatuzumab tetraxetan^§ Tigatuzumab^§ Trastuzumab^# (+deruxtecan / +emtansine) Tucotuzumab celmoleukin^§ Vandortuzumab vedotin^§ Vanucizumab^§ Veltuzumab^§ Vorsetuzumab mafodotin^§ Zenocutuzumab
Rat/mouse hybrid	Catumaxomab Ertumaxomab^§
Chimeric + humanized	Depatuxizumab mafodotin^† Duvortuxizumab Ontuxizumab^§

^#WHO-EM
^‡Withdrawn from market
Clinical trials:
- ^†Phase III
- ^§Never to phase III

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