Takeda Announces Acceptance of European Marketing Authorisation Application for Peginesatide, an Investigational Compound for the Treatment of Anemia Associated with Chronic Kidney Disease in Patients on Dialysis

LONDON and OSAKA, Japan, February 27, 2012 /PRNewswire/ --

Takeda Pharmaceutical Company Limited (Takeda) today announced that Takeda Global Research & Development Centre (Europe) Ltd received confirmation of acceptance for assessment from the European Medicines Agency (EMA) for a Marketing Authorisation Application (MAA) for the investigational compound peginesatide for the treatment of symptomatic anemia associated with chronic kidney disease (CKD) in adult patients on dialysis. Takeda had submitted the MAA earlier this month.

Peginesatide is a synthetic, PEGylated peptidic compound that binds to and activates the erythropoietin receptor and thus acts as an erythropoiesis stimulating agent (ESA) which results in the increased formation of red blood cells. The agent was discovered by Affymax, and if approved, will be co-marketed by Affymax and Takeda in the United States, and by Takeda in the European Union.

The MAA submission for peginesatide was supported mainly by data from two Phase 3 studies (EMERALD 1 and 2) that evaluated the efficacy and safety of peginesatide, dosed once every four weeks, compared to epoetin alfa or epoetin beta, dosed three times per week (according to the product labels), in maintaining hemoglobin (Hb) levels. The EMERALD studies were part of the largest Phase 3 clinical program to support the initial registration of an ESA. The program enrolled more than 2,600 patients, including approximately 1,600 dialysis patients. In the studies, CKD patients on dialysis who were receiving stable doses of epoetin, were randomized to receive once-monthly peginesatide or continue treatment with epoetin. The EMERALD findings suggested that once-monthly peginesatide was similar to epoetin in maintaining Hb levels in CKD patients on dialysis with anemia. In addition the two groups had a similar safety profile.

In May 2011, Takeda's U.S. partner, Affymax, submitted a New Drug Application for peginesatide to the U.S. Food and Drug Administration (FDA). The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 15 to 1, with 1 abstention, that peginesatide demonstrated a favorable benefit/risk profile for use in the treatment of dialysis patients with anemia due to CKD in December 2011, and a Prescription Drug User Fee Act (PDUFA) date of March 27, 2012, is scheduled for peginesatide.

"This MAA represents a significant milestone for our company and carries positive clinical implications for patients with anemia in chronic kidney disease and the physicians who treat them," said Stuart Dollow, M.D., managing director, Takeda Global Research & Development Centre (Europe) Ltd. "Although anemia is a debilitating condition for chronic kidney disease patients on dialysis, appropriate management of this condition is onerous for patients and healthcare professionals. Our program illustrates the potential of peginesatide as an important once-monthly therapeutic option. We are committed to bringing new treatments to patients with unmet needs, and look forward to working with the EMA as they review the data package for the peginesatide MAA."

^[1] National Kidney and Urologic Diseases Information Clearinghouse. "Anemia in Kidney Disease and Dialysis"

^[2] Astor et al Association of Kidney Function with Anemia: The Third National Health and Nutrition Examination Survey (1988-1992). Archives of Internal Medicine 2002; 162: 1401-1408

^[3] National Heart Lung and Blood Institute "What is Anemia." http://www.nhlbi.nih.gov/health/dci/Diseases/anemia/anemia_what_is.html.

^[4] Fishbane et al. "Hemoglobin Cycling in Hemodialysis Patients Treated With Recombinant Human Erythropoietin." Kidney International 2005; Vol. 68 (2005), pp. 1337-1343

About Anemia in Chronic Kidney Disease (CKD)

Anemia is a common complication in CKD that impacts the overall health and well-being of CKD patients and is associated with increased rates of hospitalization and mortality.^[1,2] ESAs are medications commonly prescribed to treat anemia and stimulate red blood cell production.^[3] Research has shown that there may be challenges with anemia management due to factors such as, Hb variability and stability, among other concerns.^[4]

About Peginesatide

Affymax and Takeda are collaborating on the development of peginesatide and plan to co-commercialize the product if approved in the United States. The product, upon approval, will be commercialized outside the United States by Takeda. The most common adverse events reported in the clinical studies were diarrhea, cough, dyspnea, nausea, and muscle spasm.

About Takeda Global Research & Development Centre (Europe) Ltd

Based in London, England, Takeda Global Research & Development Centre (Europe) Ltd, (TGRD Europe), is a subsidiary of Takeda Pharmaceutical Company Limited, the largest pharmaceutical company in Japan. TGRD Europe seeks to bring innovative products to patients through a pipeline that includes compounds in development for diabetes, cardiovascular disease, neurology, oncology and other conditions.

About Takeda Pharmaceutical Company Limited

Located in Osaka, Japan, Takeda is a research-based global company with its main focus on pharmaceuticals. As the largest pharmaceutical company in Japan and one of the global leaders of the industry, Takeda is committed to strive towards better health for patients worldwide through leading innovation in medicine. Additional information about Takeda is available through its corporate website, http://www.takeda.com.

Contacts:

Rebekah Childers
TGRD Europe
+44-(0)-780-949-5101

Julia Ellwanger
Takeda Pharmaceuticals International, Inc.
+1-224-554-7681

Takeda Pharmaceutical Company Limited
Corporate Communications Dept. (PR/IR)
+81-3-3278-2037