Otsuka Pharmaceutical Co., Ltd. Release: CHMP Recommends Jinarc (Tolvaptan) For Approval In EU: The First Pharmaceutical Treatment For Autosomal Dominant Polycystic Kidney Disease (Adpkd)

• JINARC^® (tolvaptan), if approved, will become the first pharmaceutical therapy available in Europe for patients with ADPKD. Tolvaptan, discovered and developed in Japan by Otsuka Pharmaceutical, was first approved there for ADPKD in 2014. It was approved for ADPKD in Canada in February 2015.
• ADPKD is a chronic and progressive genetic disease, which causes cyst growth in the kidneys, leading to an increase in kidney volume, and resulting in complications that include chronic and acute pain, hypertension and kidney failure.¹
• In a Phase III clinical trial of patients with ADPKD, tolvaptan demonstrated a statistically significant reduction of 49% in the annual increase in total kidney volume versus placebo and a reduction in the decline in kidney function by 30% versus placebo.¹
• The number of patients affected by ADPKD is estimated to be about 205,000 people in Europe.²

TOKYO--(BUSINESS WIRE)--Otsuka Pharmaceutical Co., Ltd. announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended JINARC^® (tolvaptan) for approval. This treatment has been recommended to slow the progression of cyst development and renal insufficiency of autosomal dominant polycystic kidney disease (ADPKD) in adults with chronic kidney disease (CKD) stage 1 to 3 at initiation of treatment with evidence of rapidly progressing disease.

“Otsuka strives to develop medicines that address genuine unmet medical need and we look forward to the final decision of the European Commission.”

Tolvaptan was developed over a period of 26 years through the persevering efforts of researchers in Otsuka’s Japanese pharmaceutical research centre. Upon discovering a cell signaling pathway that causes renal cysts to proliferate and enlarge,³ Otsuka launched an effort in 2004 to develop a drug for the disease in conjunction with the world’s leading ADPKD medical specialists.

The CHMP recommendation is based on data from the largest clinical study conducted in ADPKD to date – the pivotal Phase III randomised, double-blind and placebo-controlled TEMPO 3:4 trial.¹ In the three-year trial, tolvaptan achieved its primary endpoint, demonstrating a statistically significant reduction of almost half (49%) in the annual increase in total kidney volume versus placebo (p<0.001). Furthermore, the study showed tolvaptan significantly reduced the decline in kidney function by 30% versus placebo (p<0.001). The overall incidence of side effects observed in ADPKD patients administered tolvaptan in the TEMPO 3:4 trial was comparable with those administered a placebo.¹

On 5 August 2013, tolvaptan was granted orphan drug designation for the treatment of ADPKD by the European Commission.² Tolvaptan’s orphan designation highlights the EMA’s recognition of ADPKD as a rare, life-threatening and chronically debilitating condition for which there is currently no specific treatment.^2,4

“This recommendation brings us one step closer to providing to patients in Europe the first-in-the-world, disease-modifying treatment for ADPKD, a progressive and chronic genetic disease,” said Ole Vahlgren, CEO & President, Otsuka Europe. “Otsuka strives to develop medicines that address genuine unmet medical need and we look forward to the final decision of the European Commission.”

ADPKD is the most common inherited kidney disease and is primarily characterised by the development and expansion of multiple fluid-filled cysts in the kidney.^1,5 Cyst growth and expansion in both kidneys leads to slow deterioration of kidney function, and approximately half of patients reach end-stage renal disease (ESRD) and require renal replacement therapy in the form of dialysis or a kidney transplant by age 54.^4,6 ADPKD is the fourth leading cause of ESRD in adults⁷ and accounts for around 10% of patients with ESRD requiring renal replacement therapy.⁸

The European Commission (EC) reviews the recommendations of the CHMP but is not obliged to grant marketing authorisation following a recommendation for approval. A final EC decision is expected during the second quarter of 2015.

About TEMPO 3:4

The TEMPO 3:4 trial (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and its Outcomes) enrolled patients across 129 sites worldwide.¹ In this trial a total of 1,445 adult patients (age 18-50 years) with evidence of rapidly-progressing early ADPKD were enrolled between January 2007 and January 2009 and administered tolvaptan or a placebo for up to three years.

In the TEMPO 3:4 trial the risk of liver injury was identified in patients with ADPKD taking tolvaptan. Whilst this risk is small (4% in the trial), patients taking tolvaptan will have to undergo monthly blood tests for the first 18 months of treatment with tolvaptan and 3-monthly thereafter to mitigate this risk.

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 programmes 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 Pharmaceutical Europe Ltd. (OPEL) was established in 1979. Reflecting Otsuka’s ethos of ‘super people not super computers’, Otsuka’s 550 European employees focus their passion and energy into ensuring that patients in Europe have access to Otsuka’s innovative products and medical devices in our key therapy areas of CNS, endocrinology, nephrology, gastroenterology and oncology. Clinical development, commercialisation and sales and marketing operations in France, Germany, Italy, the Nordic region, Spain, Switzerland and the UK are all supported by Otsuka’s European regional office, based in west London.

Otsuka Pharmaceutical Co., Ltd. is a wholly owned subsidiary of Otsuka Holdings Co., Ltd., the holding company for the Otsuka Group. The Otsuka Group employs approximately 44,000 people globally and its products are available in more than 80 countries worldwide. Consolidated sales were over €10 billion or $14 billion for fiscal year 2013 (1 April 2013 to 31 March, 2014).

Otsuka Pharmaceutical Europe welcomes you to visit its European regional website at www.otsuka-europe.com

For more information on Otsuka Pharmaceutical Co., Ltd. please visit its global website at https://www.otsuka.co.jp/en/

References

1. Torres VE, Harris PC et al. Tolvaptan in patients with autosomal dominant polycystic kidney disease. The New England Journal of Medicine. 2012;367 (25): 2407-2418

2. European Medicines Agency. Public summary of opinion on orphan designation. 2013. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2013/09/WC500149378.pdf [Last accessed: February 2015]

3. Gattone, VH et al. Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist. Nature Medicine. 2003: 9 (10): 1323-1326

4. Takiar V, Caplan MJ. Polycystic kidney disease: pathogenesis and potential therapies. Biochimica et Biophysica Acta. 2011;1812(10):1337-43

5. Patel V, Chowdhury R et al. Advances in the pathogenesis and treatment of polycystic kidney disease. Current Opinion in Nephrology and Hypertension. 2009;18:99-106

6. Alam A, Perrone RD. Management of ESRD in Patients With Autosomal Dominant Polycystic Kidney Disease. Advances in Chronic Kidney Disease. Vol 17, No 2. March 2010: pp 164-172.

7. Masoumi A, Reed-Gitomer B et al. Developments in the Management of Autosomal Dominant Polycystic Kidney Disease. Therapeutics and Clinical Risk Management. 2008;4(2):393–407

8. Thong KM, Ong ACM. The natural history of autosomal dominant polycystic kidney disease: 30-year experience from a single centre. QJM. 2013;2-8

OPEL/0115/GEN/1006
February 2015

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