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March 31, 2014

Application Seeking Approval for Additional Indication of NESP® for Anemia with Myelodysplastic Syndrome in Japan

Tokyo, Japan, March 31, 2014 --- Kyowa Hakko Kirin Co, Ltd. (Tokyo: 4151, President and CEO: Nobuo Hanai, "Kyowa Hakko Kirin") today announced that it has filed an application to Japan's Ministry of Health, Labour and Welfare (MHLW) seeking approval for additional indication of NESP® Injection Plastic Syringe (NESP®) for anemia with myelodysplastic syndrome.

NESP®, a long-acting erythropoiesis stimulating agent, was initially approved in July 2007. NESP® is highly recognized for its safety and efficacy for treating anemia of chronic kidney disease patients on/not on dialysis and is widely used in various medical institutions.

The application seeking approval for the additional indication of NESP® has been filed based on a clinical trial conducted in Japan and Korea, as well as foreign clinical data which have provided new confirmation of its safety and efficacy for anemia with myelodysplastic syndrome. NESP® is subjected to priority review attributed to the orphan drug designation for the treatment of anemia with myelodysplastic syndrome granted in March, 2014.

Overseas guidelines for the treatment of myelodysplastic syndrome recommend the administration of darbepoetin alfa as the primary therapeutic agent for the treatment of anemia mainly in patients who have a serum erythropoietin level of equal to or less than 500 mIU/mL and are in the low risk or intermediate-1 risk categories under the International Prognostic Scoring System. However, in Japan, the use of erythropoiesis-stimulating agents including NESP® is not approved for medical treatment under health insurance, and treatment through erythrocyte transfusion is predominate. Because long-term administration of transfusions carries risks such as viral infection and hyperferrimia, the Review Committee on Unapproved or Off-Label Drugs with High Medical Needs determined that NESP® is a drug which needs to be developed as soon as possible, and Kyowa Hakko Kirin has received a request from the MHLW for its development.

Kyowa Hakko Kirin is focusing on four strategic categories including the oncology area, and is expecting to contribute to the treatment of patients with cancer through the approval of drugs in accordance with medical needs.

About myelodysplastic syndrome
Myelodysplastic syndrome is a group of diseases in which abnormalities occur in pluripotent hematopoietic stem cells in the bone marrow, and which can progress to cytopenia or acute myeloid leukemia. It is reported that there are approximately 11,000 patients in Japan with myelodysplastic syndrome, the main clinical symptoms of which are cytopenia related anemia, infection and bleeding.

About orphan drug designation
A drug must meet the following three conditions in order to be granted an orphan drug designation in Japan.

  1. The number of patients with severe disease who may use the drug is less than 50,000 in Japan.
  2. There are high medical needs for the drug (There is no appropriate alternative drug/treatment, or high efficacy or safety is expected compared with existing products).
  3. There is high possibility of development (There should be a theoretical rationale for the use of the drug for the target disease, and the development plan should be appropriate). For designated orphan drugs, measures to support the research and development activities are taken (The orphan drug and orphan medical device research and development promotion program by the MHLW).

About the International Prognostic Scoring System
The International Prognostic Scoring System is a classification system for predicting prognosis. It is considered that three factors are strongly related to the prognosis for myelodysplastic syndrome patients: (1) Percentage of immature blood cells (blasts) in the bone marrow; (2) Types of cytopenia in the peripheral blood; and (3) Types and degree of chromosomal abnormalities. By the scoring of these factors, patients are classified into one of four categories of low risk, intermediate-1 risk, intermediate-2 risk or high risk.

About the Review Committee on Unapproved or Off-Label Drugs with High Medical Needs
The Committee was established for the purpose of enhancing development by the pharmaceutical companies of drugs and indications that have been approved for use in the United States and Europe but are not yet approved in Japan, through activities such as evaluating medical needs, confirming the applicability of applications based on evidence in the public domain and investigating the need for additional studies that should be conducted.

About the four categories
The categories are comprised of the following four disease areas: Nephrology; Oncology; Immunology and Allergy; and Central Nervous System, as presented in Kyowa Hakko Kirin's FY2013-15 medium-term business plan.


Kyowa Hakko Kirin
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