{"entity":{"id":"moxetumomab-pasudotox","kind":"drug","name":"Moxetumomab pasudotox","aka":[],"tldr":"An immunotoxin for hairy cell leukaemia that produced lasting remissions in relapsed patients but was withdrawn from sale in 2023 for commercial reasons.","summary":"Moxetumomab pasudotox is a recombinant immunotoxin: an anti-CD22 Fv fragment fused to a truncated Pseudomonas exotoxin A (PE38). After binding CD22 it is internalised and inactivates elongation factor 2 by ADP-ribosylation, shutting down protein synthesis in the leukaemic cell. It was approved in the US in 2018 for relapsed or refractory hairy cell leukaemia after at least two systemic therapies including a purine analogue. The pivotal study (Kreitman, Leukemia 2018) achieved a 41% complete remission rate, 30% of them durable, in multiply relapsed patients; capillary leak and haemolytic uraemic syndrome were the key toxicities. AstraZeneca withdrew it from the US market in 2023 because of low use, with compassionate supply continuing through the NIH. It remains an example of an effective rare-disease drug that failed commercially rather than clinically.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Moxetumomab_pasudotox","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Moxetumomab_pasudotox"}],"tags":["gap-fill"],"related":[],"cancers":["hairy-cell-leukemia"],"sections":[],"technologies":["adc"],"targets":["cd22"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":["pivotal-trial"],"trials":["nct01829711","nct02338050"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lumoxiti","modality":"Anti-CD22 immunotoxin (Pseudomonas exotoxin A fragment)","mechanism":"Recombinant anti-CD22 Fv fused to truncated Pseudomonas exotoxin PE38; internalised and inhibits protein synthesis via ADP-ribosylation of EF-2.","approvals":[{"region":"US","year":2018,"indication":"Relapsed/refractory hairy cell leukaemia after ≥2 systemic therapies including a purine analogue","note":"Withdrawn from market 2023"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/moxetumomab-pasudotox/","neighbours":{"cancer":[{"id":"hairy-cell-leukemia","kind":"cancer","name":"Hairy cell leukaemia","route":"/cancers/hairy-cell-leukemia/"}],"technology":[{"id":"adc","kind":"technology","name":"Antibody-drug conjugate (ADC)","route":"/technologies/adc/"}],"target":[{"id":"cd22","kind":"target","name":"CD22","route":"/targets/cd22/"}],"company":[{"id":"astrazeneca","kind":"company","name":"AstraZeneca","route":"/companies/astrazeneca/"}],"term":[{"id":"pivotal-trial","kind":"term","name":"Pivotal (registrational) trial","route":"/terms/pivotal-trial/"}],"trial":[{"id":"nct02338050","kind":"trial","name":"Moxetumomab Pasudotox (CAT-8015, HA22) in Children With B-lineage Acute Lymphoblastic Leukemia and Minimal Residual Disease Prior to Allogeneic Hematopoietic Stem Cell Transplantation","route":"/trials/nct02338050/"},{"id":"nct01829711","kind":"trial","name":"Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia","route":"/trials/nct01829711/"}]}}