{"entity":{"id":"clofarabine","kind":"drug","name":"Clofarabine","aka":["Evoltra"],"tldr":"Clofarabine (Clolar) is an infusion for children and young adults whose acute lymphoblastic leukaemia has come back after at least two other treatments.","summary":"Clofarabine is a second-generation purine nucleoside analogue combining features of fludarabine and cladribine. The FDA granted accelerated approval in December 2004 for patients aged 1 to 21 with relapsed or refractory acute lymphoblastic leukaemia after at least two prior regimens, based on a single-arm study in which about 20% of heavily pretreated children achieved complete remission; the EU authorised Evoltra in 2006 for the same paediatric population. It is now used mainly as a bridge to transplant and in combination regimens for relapsed ALL, and off label in adult AML. Capillary leak and systemic inflammatory response syndrome, hepatotoxicity and prolonged cytopenias are the key risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Clofarabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=clofarabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/clofarabine"},{"label":"EPAR (Evoltra)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/evoltra"}],"tags":["nci-list"],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00703820","nct02883049","nct05917405"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Clolar","modality":"Purine nucleoside analogue (antimetabolite)","mechanism":"Phosphorylated by deoxycytidine kinase to clofarabine triphosphate, which inhibits ribonucleotide reductase and DNA polymerase and disrupts mitochondrial membranes, triggering apoptosis.","approvals":[{"region":"US","year":2004,"indication":"Relapsed or refractory ALL in patients aged 1 to 21 after at least two prior regimens (accelerated)"},{"region":"EU","year":2006,"indication":"Relapsed or refractory ALL in children after at least two prior regimens (Evoltra)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2004-12-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of pediatric patients 1 to 21 yrs old with relapsed or refractory acute lymphocytic leukemia (ALL) after at least 2 prior regimens"},{"date":"2022-07-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 17.6 years after it was granted.","indication":"Treatment of pediatric patients 1 to 21 yrs old with relapsed or refractory acute lymphocytic leukemia (ALL) after at least 2 prior regimens"}]},"route":"/drugs/clofarabine/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"}],"technology":[{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"}],"company":[{"id":"sanofi","kind":"company","name":"Sanofi","route":"/companies/sanofi/"}],"trial":[{"id":"nct00703820","kind":"trial","name":"Clofarabine Plus Cytarabine Versus Conventional Induction Therapy And A Study Of NK Cell Transplantation In Newly Diagnosed Acute Myeloid Leukemia","route":"/trials/nct00703820/"},{"id":"nct02883049","kind":"trial","name":"Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations","route":"/trials/nct02883049/"},{"id":"nct05917405","kind":"trial","name":"Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT","route":"/trials/nct05917405/"},{"id":"nct01700946","kind":"trial","name":"Therapy for Pediatric Relapsed or Refractory Precursor B-Cell Acute Lymphoblastic Leukemia and Lymphoma","route":"/trials/nct01700946/"}],"target":[{"id":"pola1","kind":"target","name":"DNA polymerase alpha (POLA1)","route":"/targets/pola1/"}]}}