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TMCNet:  European Medicines Agency (EMA) Accepts Otsuka's Marketing Authorisation Application (MAA) for Tolvaptan, an Investigational Compound for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

[December 27, 2013]

European Medicines Agency (EMA) Accepts Otsuka's Marketing Authorisation Application (MAA) for Tolvaptan, an Investigational Compound for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

TOKYO --(Business Wire)--

Otsuka Pharmaceutical Co., Ltd. announced today that the European Medicines Agency (EMA (News - Alert)) has accepted the submission of a marketing authorisation application (MAA) for the potential approval of tolvaptan for the treatment of autosomal dominant polycystic kidney disease (ADPKD). Phase III clinical trial results that form the basis of the regulatory filing were published in the New England Journal of Medicine.i

"Otsuka is delighted that the EMA will review the tolvaptan MAA for the treatment of ADPKD, based on compelling data from our pivotal three-year Phase III clinical trial," said Ole Vahlgren, CEO & President, Otsuka Europe. "If approved, tolvaptan will represent a breakthrough for patients with a disease for which there are currently no licensed treatments."

Tolvaptan is a selective V2 vasopressin receptor antagonist that has been hypothesised to slow the progression of ADPKD by reducing the development and growth of kidney cysts.

ADPKD is a hereditary genetic disease characterised by the development of multiple, non-malignant cysts in the kidneys due to inherited or acquired genetic mutation(s).ii,iii,iv Cyst growth and expansion in both kidneys leads to slow deterioration of kidney function, and in approximately 50% of patients, to end-stage renal disease (ESRD) and renal failure.v ADPKD typically results in symptom manifestations (e.g. hypertension and kidney pain) in adulthood.v Half of all ADPKD patients will require dialysis or transplantation by the age of 60 and people with ADPKD account for up to 1 in 10 people on maintenance dialysis.vi,vii The condition is estimated to affect approximately 200,000 people in Europe.ii

The Committee for Medicinal Products for Human Use (CHMP), a committee within the EMA, is responsible for evaluating the application and will provide a recommendation on whether tolvaptan should receive marketing authorisation. The CHMP may request further information from the applicant before adopting an opinion. If the committee's opinion is positive, it is forwarded to the European Commission to make the final decision.

In August 2013, the EMA's Committee for Orphan Medicinal Products (COMP) grnted tolvaptan orphan drug designation for the treatment of ADPKD.ii To qualify for orphan designation, the prevalence of the condition in the EU must not be more than 5 in 10,000. In addition, the new medicine must be of significant additional benefit to those affected by the condition, and no satisfactory method of diagnosis, prevention or treatment of the condition must exist.


In Japan, the application for ADPKD is currently under review. In the United States, Otsuka and FDA have been working together to determine the most appropriate path forward to allow tolvaptan to be available for patients suffering from ADPKD.

About Otsuka Pharmaceutical Co., Ltd.

Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy: 'Otsuka-people creating new products for better health worldwide.' Otsuka researches, develops, manufactures and markets innovative and original products, with a focus on pharmaceutical products for the treatment of diseases and nutraceutical products for the maintenance of everyday health.

In pharmaceuticals, Otsuka is a leading firm in the challenging area of mental health and also has research programs for several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate more powerfully than words how Otsuka is a "big venture" company at heart, applying a youthful spirit of creativity in everything it does.

Otsuka is a wholly owned subsidiary of Otsuka Holdings Co., Ltd., the holding company for the Otsuka Group. The chairman Akihiko Otsuka is the third generation of Otsuka family members to lead the business, whose origins date from 1921. The Otsuka Group employs approximately 42,000 people globally and its products are available in more than 80 countries worldwide. Consolidated sales were approximately €10 billion or USD 13 billion for fiscal year 2012 (4/1/2012-3/31/2013). Otsuka Pharmaceutical welcomes you to visit its global website at http://www.otsuka.co.jp/en/

i Torres VE, Chapman AB, et al. Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease. New England Journal of Medicine. 2012;367:2407-2418.

ii European Medicines Agency. EU/3/13/1175. 2013. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2013/09/human_orphan_001257.jsp&mid=WC0b01ac058001d12b [Last accessed: December 2013]

iii Torrres VE, Harris PC, et al. Autosomal dominant polycystic kidney disease. Lancet. 2007; 369: 1287-1301

iv Tan Y, Blumenfeld J and Rennert H. Autosomal dominant polycystic kidney disease: genetics, mutations and microRNA. Biochimica Biophysica Acta. 2011; 1812: 1202-1212

v Patel V, Chowhury R, and Igarashi P. Advances in the pathogenesis and treatment of polycystic kidney disease. Current Opinion in Nephrology and Hypertension. 2009;18:99-106.

vi Renal Resource Centre. Polycystic Kidney Disease. Available from: http://www.renalresource.com/booklets/pkd.php [last accessed December 2013]

vii Christophe JL, van Ypersele de Strihou C, et al. Complications of autosomal dominant polycystic kidney disease in 50 haemodialysed patients. A case-control study. Nephrology. Dialysis. Transplantation 1996; 11:1271-1276.


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