NEWS RELEASE
March 26, 2024

Vyxeos® Combination for I.V. Injection Receives Approval for the Treatment ofHigh-risk AML in Japan
Kyoto, Japan, March 26, 2024 - Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters,Kyoto; President, Toru Nakai) announced today that the Ministry of Health, Labour and Welfare(MHLW)

hasapproved

Vyxeos®

CombinationforI.V.

Injection(Daunorubicinhydrochloride/cytarabine liposomal drug for injection) for the treatment of high-risk acute myeloid
leukemia (AML) in Japan.
AML is a disease of clonal proliferation of immature myeloid cells, and results from genetic
abnormalities in myeloblasts which are immature blood cells. The uncontrollable growth of leukemia
cells in the bone marrow prevents the production of normal blood which results in various symptoms
such as infections and bleeding.1 Although multiple AML drugs have been launched in recent years
and the outcomes for some patient groups have improved, many patients still do not have longterm survival. Novel therapeutic agents with greater efficacy are needed.2
Vyxeos® is a liposomal formulation of a fixed combination of cytarabine and daunorubicin, thestandard drugs for AML treatment, at 5:1 molar ratio. The 5:1 molar ratio has been shown tomaximize synergistic antitumor activity in AML. After the drug is taken up by leukemia cells in thebone marrow, cytarabine and daunorubicin are released to exert its anti-tumor effect.
Nippon Shinyaku acquired a license for Vyxeos® from Jazz Pharmaceuticals plc (Headquarters:Dublin, Ireland, Chairman and CEO: Bruce C. Cozadd) on March 30, 2017, and has beendeveloping it in Japan. The approval is based on data from a Phase I/II study conducted by Nippon
Shinyaku in Japan and a Phase III study conducted by Jazz in overseas for patients with high-risk
AML*, which is considered to have poorer prognosis compared to other forms of AML. The Phase
III study met its primary endpoint, as Vyxeos demonstrated a superior improvement in overall
survival compared to the standard of care 7+3 treatment regimen for the first time. The medianoverall survival for the Vyxeos treatment group was 9.56 months compared with 5.95 months forthe 7+3 treatment group (1-sided p-value = 0.003; HR [95% confidence interval] = 0.69 [0.52,0.90]).3
Vyxeos® is approved for the treatment of therapy-related AML and AML with myelodysplasticchanges in more than 30 countries or regions around the world, including Europe and the UnitedStates. Vyxeos® was designated as an orphan drug by MHLW for the indication of AML in May
2022.

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Nippon Shinyaku believes that it will contribute to the treatment of high-risk AML by delivering
Vyxeos® appropriately to healthcare professionals and their patients who need it.
* The definition of high-risk AML is if any of the following conditions below apply.
・therapy-related AML・AML with a history of myelodysplastic syndromes (MDS)・de novo AML with MDS-related cytogenetic abnormalities
・AML with a history of chronic myelomonocytic leukemia (CMML)

References
1. Japanese Society of Hematology. Practical Guidelines for Hematological Malignancies, 2023,
Kanehara & Co., Ltd. (Japanese only).
2. Gurnari C et al. Deciphering the Therapeutic Resistance in Acute Myeloid Leukemia. Int J Mol
Sci. 2020; 21(22):8505.
3. Lancet JE et al. CPX-351 (cytarabine and daunorubicin) Liposome for Injection versus
Conventional Cytarabine Plus Daunorubicin in Older Patients with Newly Diagnosed
Secondary Acute Myeloid Leukemia. J Clin Oncol. 2018; 36(26):2684-2692.
Summary of Vyxeos®
Brand name
Generic name
Dosage Forms and Strengths
Indication

Dosage and Administration

Vyxeos® Combination for I.V. Injection

Daunorubicin hydrochloride/cytarabine liposomal drug for injection
Freeze-dried preparation for injection containing 47mg of daunorubicinhydrochloride (44mg as daunorubicin) and 100mg of cytarabine
High-risk acute myeloid leukemia(1) Remission induction therapy
Usually, as remission induction therapy, 100 units (44mg as
daunorubicin and 100mg as cytarabine)/m 2 (body surface area) ofVyxeos® Combination for I.V. Injection shall be administered over 90
minutes once daily for up to two cycles.
This drug shall be intravenously administered by drip infusion on Days
1, 3, and 5 of Cycle 1. This drug shall be intravenously administered bydrip infusion on Days 1 and 3 of Cycle 2 to patients who do not achieveremission during Cycle 1, if there are no unacceptable toxicities, two tofive weeks after the start of Cycle 1.
(2) Consolidation therapy
Usually, as consolidation therapy, 65 units (29mg as daunorubicin and65mg as cytarabine)/m 2 (body surface area) of Vyxeos® Combination forI.V. Injection shall be administered over 90 minutes once daily for up totwo cycles.
This drug shall be intravenously administered by drip infusion on Days
1 and 3 of Cycle 1, five to eight weeks after the start of the last remission
induction therapy. This drug shall be intravenously administered by drip
infusion on Days 1 and 3 of Cycle 2 to patients without diseaseprogression after the start of administration of Cycle 1, if there are nounacceptable toxicities, five to eight weeks after the start of Cycle 1 ofthe consolidation therapy.
One dose unit of this drug contains 0.44mg of daunorubicin and 1mg ofcytarabine.

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About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,”
we aim to be an organization trusted by the community through creating unique medicines that willbring hope to patients and families suffering from illness. Please visit our website(https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.
Contact
Corporate Communications Dept., Nippon Shinyaku Co., Ltd.
FAX: +81-75-321-9128

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