Leukaemia therapy has become increasingly targeted and molecularly defined, with new drug classes reaching approval across the myeloid and lymphoid leukaemias.
Chronic myeloid leukaemia
Asciminib (Scemblix) introduced a new mechanism to CML — it binds the myristoyl pocket of BCR-ABL (a "STAMP" allosteric inhibitor) rather than the ATP site, giving it activity against resistant disease (including the T315I mutation) and better tolerability. Approved first for later lines, it gained a frontline indication in 2024 (the ASC4FIRST trial), expanding options beyond the traditional ATP-competitive tyrosine kinase inhibitors.
Acute lymphoblastic leukaemia
Immunotherapy has moved to the front of B-cell ALL treatment. Brexucabtagene autoleucel (Tecartus), a CD19 CAR T-cell therapy, is approved for relapsed/refractory B-ALL, and the CD19 bispecific T-cell engager blinatumomab — long used in relapsed and minimal-residual-disease-positive settings — has moved into frontline consolidation after a trial showed it improves overall survival when added to chemotherapy in newly diagnosed patients. Chemotherapy-sparing combinations (such as a TKI plus blinatumomab for Philadelphia-chromosome-positive ALL) are also advancing.
Acute myeloid leukaemia
AML care is now strongly genotype-driven. Venetoclax plus a hypomethylating agent transformed outcomes for older and unfit patients; FLT3 inhibitors (midostaurin and quizartinib frontline, gilteritinib in relapse) target FLT3-mutated disease; and IDH inhibitors (ivosidenib and olutasidenib for IDH1, enasidenib for IDH2) address those mutations, with differentiation syndrome a shared class effect.
The newest class is the menin inhibitors. Revumenib (Revuforj), the first-in-class agent, was approved in November 2024 for relapsed or refractory KMT2A-rearranged acute leukaemia and expanded in October 2025 to relapsed or refractory NPM1-mutated AML — the single most common AML mutation, present in a large share of cases. This is a landmark, providing the first targeted therapy for aggressive subtypes that were previously untargetable, and additional menin inhibitors are following. Established options — gemtuzumab ozogamicin, CPX-351 for secondary AML, oral azacitidine maintenance, and glasdegib — round out the toolkit, while CD33 CAR-T and CD3×CD123 bispecific approaches remain investigational.
Further reading: asciminib (Scemblix) trials and ASC4FIRST frontline approval; venetoclax-azacitidine VIALE-A; quizartinib QuANTUM-First; revumenib AUGMENT-101 (Issa et al., JCO 2025) and 2024–2025 approvals; AML management reviews (Kantarjian et al., CA Cancer J Clin 2025).