Kyowa Hakko Kirin announced that is seeking approval from the Japanese Ministry of Health, Labor, and Welfare (MHLW) for additional indication of NESP Injection Plastic Syringe (NESP) for anemia with myelodysplastic syndrome.
NESP is a long-acting erythropoiesis stimulating agent initially approved in July 2007. The drug has established its safety and efficacy profile in the treatment of anemia of chronic kidney disease patients with or without dialysis.
“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,” Kyowa stated in its press release.
Overseas guidelines recommend for the administration of darbepoetin alfa as treatment of myelodysplastic syndrome. However, treatment through erythrocyte transfusion predominates in Japan and the use of erythropoiesis-stimulating agents, including NESP, is currently not approved for medical treatment under health insurance policy.
Myelodysplastic syndrome is a group of diseases wherein abnormalities occur in pluripotent hematopoietic stem cells in the bone marrow. This can progress to acute myeloid leukemia or cytopenia. Main clinical symptoms of the disease include cytopenia related anemia, infection, and bleeding. An estimated 11,000 people in Japan are affected with myelodysplastic syndrome.
As standard treatment carries risks, such as viral infection and hyperferrimia, the company has received a request from the MHLW for NESP’s development. “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,” Kyowa stated. The Review Committee helps drug makers develop drugs which are approved for use in the United States and Europe, but not in Japan.