{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T16:30:36Z","timestamp":1792513836062,"version":"4.5.0"},"reference-count":9,"publisher":"Oxford University Press (OUP)","issue":"9","license":[{"start":{"date-parts":[[2020,4,13]],"date-time":"2020-04-13T00:00:00Z","timestamp":1586736000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020,9,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title\/>\n                    <jats:p>On June 28, 2018, the Committee for Medicinal Products for Human Use adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Vyxeos, intended for the treatment of acute myeloid leukemia (AML). Vyxeos was designated as an orphan medicinal product on January 11, 2012. The applicant for this medicinal product was Jazz Pharmaceuticals Ireland Limited. Vyxeos is a liposomal formulation of a fixed combination of daunorubicin and cytarabine, antineoplastic agents that inhibit topoisomerase II activity and also cause DNA damage. The strength of Vyxeos is 5 units\/mL, where 1 unit equals 1.0 mg cytarabine plus 0.44 mg daunorubicin. The marketing authorization holder Jazz Pharmaceuticals had found that this was an optimal ratio for the efficacy of the product. Study CLTR0310-301, a phase III, multicenter, randomized, trial of Vyxeos (daunorubicin-cytarabine) liposome injection versus standard 3+7 daunorubicin and cytarabine in patients aged 60\u201375\u2009years with untreated high-risk (secondary) AML, showed a statistically significant difference between the two groups in overall survival (OS) with a median OS of 9.56 months in the daunorubicin-cytarabine arm compared with 5.95 months for standard chemotherapy (hazard ratio, 0.69; 95% confidence interval, 0.52\u20130.90; one-sided p = .003).<\/jats:p>\n                    <jats:p>The most common side effects were hypersensitivity including rash, febrile neutropenia, edema, diarrhea\/colitis, mucositis, fatigue, musculoskeletal pain, abdominal pain, decreased appetite, cough, headache, chills, arrhythmia, pyrexia, sleep disorders, and hypotension.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Implications for Practice<\/jats:title>\n                    <jats:p>Vyxeos has demonstrated a clinically significant improvement in overall survival compared with the standard of care 7+3 in the proposed population of patients with newly diagnosed acute myeloid leukemia (AML) with myelodysplasia-related changes and therapy-related AML. This is remarkable given the very poor prognosis of these patients and their unmet medical need. Secondary endpoints support the primary outcome, in particular an increased rate of hematopoietic stem cell transplantation, which is potentially the only curative treatment in AML.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1634\/theoncologist.2019-0785","type":"journal-article","created":{"date-parts":[[2020,4,13]],"date-time":"2020-04-13T08:34:44Z","timestamp":1586766884000},"page":"e1414-e1420","source":"Crossref","is-referenced-by-count":80,"title":["EMA Review of Daunorubicin and Cytarabine Encapsulated in Liposomes (Vyxeos, CPX-351) for the Treatment of Adults with Newly Diagnosed, Therapy-Related Acute Myeloid Leukemia or Acute Myeloid Leukemia with Myelodysplasia-Related Changes"],"prefix":"10.1093","volume":"25","author":[{"given":"Kyriaki","family":"Tzogani","sequence":"first","affiliation":[{"name":"European Medicines Agency, Amsterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karri","family":"Penttil\u00e4","sequence":"additional","affiliation":[{"name":"Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands"},{"name":"Finnish Medicines Agency, Fimea, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tuomo","family":"Lapvetel\u00e4inen","sequence":"additional","affiliation":[{"name":"Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands"},{"name":"Finnish Medicines Agency, Fimea, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Hemmings","sequence":"additional","affiliation":[{"name":"Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands"},{"name":"Medicines and Healthcare Products Regulatory Agency, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janet","family":"Koenig","sequence":"additional","affiliation":[{"name":"Committee for Medicinal Products for Human Use (CHMP), Amsterdam, The Netherlands"},{"name":"Federal Institute for Drugs and Medical Devices, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jo\u00e3o","family":"Freire","sequence":"additional","affiliation":[{"name":"Pharmacovigilance Risk Assessment Committee (PRAC), Amsterdam, The Netherlands"},{"name":"INFARMED, IP, Lisboa, Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silva","family":"M\u00e1rcia","sequence":"additional","affiliation":[{"name":"Pharmacovigilance Risk Assessment Committee (PRAC), Amsterdam, The Netherlands"},{"name":"INFARMED, IP, Lisboa, Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Cole","sequence":"additional","affiliation":[{"name":"Committee for Medicinal Products 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