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.