Date: 2017-03-30
Type of information: Licensing agreement
Compound: Defitelio® (defibrotide sodium), Vyxeos® (cytarabine and daunorubicin liposome injection)
Company: Jazz Pharmaceuticals (Ireland) Nippon Shinyaku (Japan)
Therapeutic area: Cancer - Oncology - Rare diseases
Type agreement: development - commercialisation
Action mechanism: antimetabolite/antimitotic agent/cytotoxic agent/ oligonucleotide. Cytarabine is an antimetabolite antineoplastic agent that inhibits the synthesis of DNA. Its actions are specific for the S phase of the cell cycle. Daunorubicin has antimitotic and cytotoxic activity. It forms complexes with DNA by intercalation between base pairs. It inhibits topoisomerase II activity by stabilizing the DNA-topoisomerase II complex, preventing the religation portion of the ligation-religation reaction that topoisomerase II catalyzes. Vyxeos™ (cytarabine and daunorubicin liposome injection), or CPX-351, is a combination of cytarabine and daunorubicin encapsulated within a nano-scale liposome at a 5:1 molar ratio. Vyxeos was granted orphan drug status by the FDA and the European Commission for the treatment of acute myeloid leukemia. Vyxeos™ was granted Breakthrough Therapy Designation for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes and was also granted Fast Track designation by the FDA for the treatment of older patients with secondary AML. Jazz Pharmaceuticals has initiated a rolling NDA submission to the FDA with expected completion of the submission by the end of March 2017 . A Marketing Authorization Application to the European Medicines Agency is planned for the second half of 2017. There have been no studies with CPX-351 in Japan , and the Vyxeos trade name has not been approved. Defibrotide is the sodium salt of a complex mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA. It has protective effects on vascular endothelial cells, particularly those of small vessels. It has extensive beneficial pharmacological effects owing to its antithrombotic, anti-inflammatory and antiischemic properties. In 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients undergoing HSCT therapy. Defibrotide is also being investigated in a global Phase 3 randomized study (NCT02851407) for the prevention of hepatic VOD in high risk adult and pediatric patients undergoing HSCT. Defibrotide is currently an investigational drug in Japan and the Defitelio trade name has not been approved.
Disease: acute myeloid leukemia, adult and pediatric patients with hepatic veno-occlusive disease (VOD), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)
Details: • On March 30, 2017, Jazz Pharmaceuticals announced that it has entered into license agreements with Nippon Shinyaku for Defitelio® (defibrotide sodium) and Vyxeos™ (cytarabine and daunorubicin liposome injection), or CPX-351, in Japan . Under the terms of the agreements, Nippon Shinyaku will receive exclusive rights to develop and commercialize Defitelio and Vyxeos in Japan in return for an upfront payment to Jazz Pharmaceuticals and subsequent payments based on the successful achievement of certain regulatory and commercial milestones. Jazz Pharmaceuticals will manufacture and supply Defitelio and Vyxeos to Nippon Shinyaku , and will receive revenue based on a percentage of product sales in Japan. Commercialization of Defitelio and Vyxeos in Japan is subject to regulatory approval in Japan .
Financial terms: Financial terms of the agreement have not been disclosed.
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