ATC Group: L01XX Other antineoplastic agents

The World Health Organization's ATC classification organizes medical drugs based on therapeutic properties, chemical composition, and anatomy. It helps make essential medicines readily available globally and is widely used in the pharmaceutical industry.

Position of L01XX in the ATC hierarchy

Level Code Title
1 L Antineoplastic and immunomodulating agents
2 L01 Antineoplastic agents
3 L01X Other antineoplastic agents
4 L01XX Other antineoplastic agents

Group L01XX contents

Code Title
L01XX01 Amsacrine
L01XX02 Asparaginase
L01XX03 Altretamine
L01XX05 Hydroxycarbamide
L01XX07 Lonidamine
L01XX08 Pentostatin
L01XX10 Masoprocol
L01XX11 Estramustine
L01XX16 Mitoguazone
L01XX18 Tiazofurine
L01XX23 Mitotane
L01XX24 Pegaspargase
L01XX27 Arsenic trioxide
L01XX29 Denileukin diftitox
L01XX33 Celecoxib
L01XX35 Anagrelide
L01XX36 Oblimersen
L01XX40 Omacetaxine mepesuccinate
L01XX41 Eribulin

Active ingredients in L01XX

Active Ingredient Description

Adagrasib is a selective, irreversible inhibitor of KRAS (Kirsten rat sarcoma viral oncogene homolog) G12C that covalently binds to the mutant cysteine in KRAS G12C and locks the mutant KRAS protein in its inactive, GDP-bound conformation, which prevents KRAS-dependent downstream signalling. Adagrasib inhibits tumour cell growth and viability in cells harbouring KRAS G12C mutations and results in regression in KRAS G12C-positive nonclinical tumour models with minimal off-target activity.


Aflibercept, also known as VEGF TRAP in the scientific literature, is a recombinant fusion protein consisting of VEGF-binding portions from the extracellular domains of human VEGF receptors 1 and 2 fused to the Fc portion of the human IgG1. Aflibercept blocks the activation of VEGF receptors and the proliferation of endothelial cells, thereby inhibiting the growth of new vessels that supply tumours with oxygen and nutrients.


Altretamine is an antineoplastic agent. It is indicated for use as a single agent in the palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy with cisplatin and/or alkylating agent-based combination. It is not considered a first-line treatment, but it can be useful as salvage therapy.

Acridinyl Anisidide

Amsidine is a sterile antitumour chemotherapeutic agent for intravenous infusion. Although not completely clarified, the mode of action of amsacrine is related to its property of binding the DNA through intercalation and external (electrostatic) forces.


Anagrelide is an inhibitor of cyclic AMP phosphodiesterase III and suppress expression of transcription factors including GATA-1 and FOG-1 required for megakaryocytopoiesis, ultimately leading to reduced platelet production.

Arsenic trioxide

Arsenic trioxide causes morphological changes and deoxyribonucleic acid (DNA) fragmentation characteristic of apoptosis in NB4 human promyelocytic leukaemia cells in vitro. Arsenic trioxide also causes damage or degradation of the fusion protein PML/RAR alpha.


Asparaginase hydrolyses asparagine to aspartic acid and ammonia. In contrast to normal cells, lymphoblastic tumour cells have a very limited capacity for synthesising asparagine because of a significantly reduced expression of asparagine synthetase. Therefore, they require asparagine which diffuses from the extracellular environment. As a result of asparaginase-induced asparagine depletion in serum, protein synthesis in lymphoblastic tumour cells is disturbed while sparing most normal cells.


Belzutifan is an inhibitor of hypoxia-inducible factor 2 alpha (HIF-2α). Belzutifan binds to HIF-2α, and in conditions of hypoxia or impairment of VHL protein function, belzutifan blocks the HIF-2α-HIF-1β interaction, leading to reduced transcription and expression of HIF-2α target genes. In vivo, belzutifan demonstrated anti-tumor activity in mouse xenograft models of renal cell carcinoma.


Celecoxib is an oral, selective, cyclooxygenase-2 (COX-2) inhibitor within the clinical dose range (200-400 mg daily). No statistically significant inhibition of COX-1 (assessed as ex vivo inhibition of thromboxane B2 [TxB2] formation) was observed in this dose range in healthy volunteers.

Denileukin diftitox

Denileukin diftitox is a recombinant DNA-derived cytotoxic protein composed of the amino acid sequences for diphtheria toxin fragments A and B (Met1 –Thr387)-His and the sequences for human interleukin-2 (IL-2; Ala1 –Thr133).


Eflornithine irreversibly inhibits ornithine decarboxylase, an enzyme involved in the production of the hair shaft by the hair follicle. Eflornithine has been shown to reduce the rate of hair growth.


Enasidenib is a small molecule inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme. Enasidenib targets the mutant IDH2 variants R140Q, R172S, and R172K at approximately 40-fold lower concentrations than the wild-type enzyme in vitro. Inhibition of the mutant IDH2 enzyme by enasidenib led to decreased 2-hydroxyglutarate (2-HG) levels and induced myeloid differentiation in vitro and in vivo in mouse xenograft models of IDH2 mutated AML. In blood samples from patients with AML with mutated IDH2, enasidenib decreased 2-HG levels, reduced blast counts and increased percentages of mature myeloid cells.

Eribulin mesylate

Eribulin is a microtubule dynamics inhibitor belonging to the halichondrin class of antineoplastic agents. It is a structurally simplified synthetic analogue of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai.


Estramustine is a chemical compound of oestradiol and nitrogen mustard. It is effective in the treatment of advanced prostatic carcinoma. Estramustine has a dual mode of action. The intact molecule acts as an anti-miotic agent; after hydrolysis of the carbamate ester, the metabolites act to bridge the released oestrogens and exert an anti-gonadotrophic effect.


One of the mechanisms by which hydroxycarbamide acts is the elevation of foetal haemoglobin (HbF) concentrations in sickle cell patients. In addition hydroxycarbamide causes an immediate inhibition of DNA synthesis by acting as a ribonucleotide reductase inhibitor, without interfering with the synthesis of ribonucleic acid or protein.


Imetelstat is an oligonucleotide human telomerase inhibitor that binds to the template region of the RNA component of human telomerase (hTR), inhibits telomerase enzymatic activity and prevents telomere binding. Increased telomerase activity and human telomerase reverse transcriptase (hTERT) RNA expression have been reported in MDS and malignant stem and progenitor cells.


Ivosidenib is an inhibitor of the mutant IDH1 enzyme. Mutant IDH1 converts alpha- ketoglutarate (αKG) to 2-hydroxyglutarate (2-HG) which blocks cellular differentiation and promotes tumorigenesis in both hematologic and non-hematologic malignancies. The mechanism of action of ivosidenib beyond its ability to reduce 2-HG and restore cellular differentiation is not fully understood across indications.


Mitotane is an adrenal cytotoxic active substance, although it can apparently also cause adrenal inhibition without cellular destruction. Available data suggest that mitotane modifies the peripheral metabolism of steroids and that it also directly suppresses the adrenal cortex. The administration of mitotane alters the extra-adrenal metabolism of cortisol in humans, leading to a reduction in measurable 17-hydroxy corticosteroids, even though plasma levels of corticosteroids do not fall.


Nirogacestat is a gamma secretase inhibitor that blocks proteolytic activation of the Notch receptor. When dysregulated, Notch can activate pathways that contribute to tumor growth.

Omacetaxine mepesuccinate

Omacetaxine mepesuccinate is a cephalotaxine ester. The mechanism of action of omacetaxine mepesuccinate has not been fully elucidated but includes inhibition of protein synthesis and is independent of direct Bcr-Abl binding. It is used for the treatment of patients with chronic or accelerated phase chronic myeloid leukemia (CML) with resistance and/or intolerance to two or more tyrosine kinase inhibitors (TKI).


Pegaspargase is a covalent conjugate of Escherichia coli-derived L-asparaginase with monomethoxypolyethylene glycol. The mechanism of action of L-asparaginase is the enzymatic cleavage of the amino acid L-asparagine into aspartic acid and ammonia. Depletion of L-asparagine in blood results in inhibition of protein-synthesis, DNA-synthesis and RNA-synthesis, especially in leukaemic blasts which are not able to synthesise L-asparagine, thus undergoing apoptosis.


Pentostatin is a potent transition state inhibitor of the enzyme adenosine deaminase. The greatest activity of ADA is found in cells of the lymphoid system with T-cells having higher activity than B-cells and T-cell malignancies higher ADA activity than B-cell malignancies. Pentostatin has been shown to have activity against a variety of lymphoid malignancies, but is most active against indolent cancers with lower ADA concentration, such as hairy cell leukaemia.


Selinexor is a nuclear export inhibitor. In nonclinical studies, selinexor reversibly inhibits nuclear export of tumor suppressor proteins (TSPs), growth regulators, and mRNAs of oncogenic proteins by blocking exportin 1 (XPO1).


Sotorasib is a selective KRAS G12C (Kirsten rat sarcoma viral oncogene homolog) inhibitor, which covalently and irreversibly binds to the unique cysteine of KRAS G12C. Inactivation of KRAS G12C by sotorasib blocks tumour cell signalling and survival, inhibits cell growth, and promotes apoptosis selectively in tumours harbouring KRAS G12C, an oncogenic driver of tumourigenesis.


Tagraxofusp is a CD123-directed cytotoxin composed of recombinant human interleukin-3 (IL-3) and truncated diphtheria toxin (DT) fusion protein that inhibits protein synthesis and causes cell death in CD123-expressing cells. It is used for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).


Tebentafusp is a bispecific fusion protein, comprised of a T cell receptor (TCR; targeting domain) fused to an antibody fragment targeting CD3 (cluster of differentiation 3; effector domain). The TCR end binds with high affinity to a gp100 peptide presented by human leukocyte antigen – A*02:01 (HLA-A*02:01) on the cell surface of uveal melanoma tumour cells, and the effector domain binds to the CD3 receptor on the polyclonal T cell.


Venetoclax is a potent, selective inhibitor of B-cell lymphoma (BCL)-2, an anti-apoptotic protein. Overexpression of BCL-2 has been demonstrated in CLL cells where it mediates tumour cell survival and has been associated with resistance to chemotherapeutics. Venetoclax binds directly to the BH3-binding groove of BCL-2, displacing BH3 motif-containing pro-apoptotic proteins like BIM, to initiate mitochondrial outer membrane permeabilization (MOMP), caspase activation, and programmed cell death.

Related product monographs

Title Information Source Document Type  
AGRYLIN Capsule FDA, National Drug Code (US) MPI, US: SPL/PLR
AMSIDINE 50mg/ml Concentrate and solvent for solution for infusion Health Products Regulatory Authority (IE) MPI, EU: SmPC
ELZONRIS Solution for injection FDA, National Drug Code (US) MPI, US: SPL/PLR
EMCYT Capsule FDA, National Drug Code (US) MPI, US: SPL/Old
ENRYLAZE Solution for injection/infusion European Medicines Agency (EU) MPI, EU: SmPC
ESTRACYT Capsule Medicines & Healthcare Products Regulatory Agency (GB) MPI, EU: SmPC
HALAVEN Solution for injection European Medicines Agency (EU) MPI, EU: SmPC
HYDREA Capsule FDA, National Drug Code (US) MPI, US: SPL/PLR
IDHIFA Film-coated tablet FDA, National Drug Code (US) MPI, US: SPL/PLR
KIDROLASE Solution for injection Health Products and Food Branch (CA) MPI, CA: SPM
KIMMTRAK Concentrate for solution for infusion European Medicines Agency (EU) MPI, EU: SmPC
KRAZATI Film-coated tablet European Medicines Agency (EU) MPI, EU: SmPC
LUMYKRAS Film-coated tablet European Medicines Agency (EU) MPI, EU: SmPC
LYSODREN Tablet European Medicines Agency (EU) MPI, EU: SmPC
MEVLYQ Solution for injection European Medicines Agency (EU) MPI, EU: SmPC
NIPENT Powder for solution for injection/infusion Medicines & Healthcare Products Regulatory Agency (GB) MPI, EU: SmPC
ONCASPAR Solution for injection/infusion European Medicines Agency (EU) MPI, EU: SmPC
RYLAZE Solution for injection FDA, National Drug Code (US) MPI, US: SPL/PLR
RYTELO Solution for injection FDA, National Drug Code (US) MPI, US: SPL/PLR
SIKLOS Film-coated tablet European Medicines Agency (EU) MPI, EU: SmPC
TAZVERIK Film coated tablet FDA, National Drug Code (US) MPI, US: SPL/PLR
TIBSOVO Film-coated tablet FDA, National Drug Code (US) MPI, US: SPL/PLR
TIBSOVO Film-coated tablet European Medicines Agency (EU) MPI, EU: SmPC
TRISENOX Concentrate for solution for infusion European Medicines Agency (EU) MPI, EU: SmPC
VENCLEXTA Film coated tablet FDA, National Drug Code (US) MPI, US: SPL/PLR
VENCLYXTO Film-coated tablet European Medicines Agency (EU) MPI, EU: SmPC
WELIREG Film-coated tablet FDA, National Drug Code (US) MPI, US: SPL/PLR
XAGRID Capsule European Medicines Agency (EU) MPI, EU: SmPC
XROMI Oral solution European Medicines Agency (EU) MPI, EU: SmPC
ZALTRAP Concentrate for solution for infusion European Medicines Agency (EU) MPI, EU: SmPC
ZEPZELCA Powder for solution for injection FDA, National Drug Code (US) MPI, US: SPL/PLR