Otsuka Announces Positive Phase III Data on Tolvaptan, in Development in U.S. for ADPKD

  • Trial achieved primary and secondary endpoints in an additional Phase III clinical trial that examined the efficacy and safety of tolvaptan in autosomal dominant polycystic kidney disease (ADPKD)
  • Aim is for data to support a full review by the FDA of a New Drug Application (NDA) for tolvaptan in ADPKD
  • Trial results will be presented at a nephrology medical congress during 2017


 TOKYO, JAPAN – May XX, 2017  Otsuka Pharmaceutical Co., Ltd. announces positive top-line results from a Phase III clinical trial of tolvaptan in adult patients with ADPKD.  

The primary endpoint of the trial was the reduction in the change of the estimated glomerular filtration rate (eGFR) from pre-treatment baseline to post-treatment assessment. Patients treated with tolvaptan showed a XX reduction in eGFR compared to a YY reduction in patients who received a placebo. GFR is an estimate of the sum of the filtration rates of all the single functional nephrons in the kidneys, measured through creatine-based estimation equations.

The secondary trial endpoint was a comparison of the efficacy of tolvaptan treatment versus placebo in reducing the decline of annualized eGFR slope.

The trial was completed with the aim of using the data to support a full review by the U.S. Federal Drug Administration (FDA) of a New Drug Application (NDA) for tolvaptan in the treatment of adults with ADPKD.In August 2013, the FDA issued a Complete Response Letter indicating that it could not accept the NDA as submitted.

The Phase IIIb, multicenter, international, randomized-withdrawal, placebo-controlled, double-blind trial compared the efficacy and safety of tolvaptan (45 to 120 mg/day) to placebo. Trial enrollees were adults (age 18 to 66) with chronic kidney disease between late stage 2 to early stage 4 due to ADPKD and not previously treated with tolvaptan. Approximately 1,370 patients were enrolled and assigned to either the tolvaptan or placebo group for a 12-month trial period.

There were no new safety issues identified in tolvaptan during the trial.

Results from the trial will be presented at a nephrology medical congress during 2017.

About
The formation of cysts associated with ADPKD can lead to significant kidney damage, even when kidney function is not affected and cause complications that include chronic and acute pain, hypertension and kidney failure.1,[3] In addition to the physical symptoms, ADPKD causes a profound psychological and emotional burden for those living with the disease, their families and loved ones.[4], [5],

About Otsuka
Otsuka Pharmaceutical 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 to meet unmet medical needs and nutraceutical products for the maintenance of everyday health.

In pharmaceuticals, Otsuka is a leader in the challenging area of mental health and also has research programs on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does.

Otsuka Pharmaceutical is a subsidiary of Otsuka Holdings Co., Ltd., headquartered in Tokyo, Japan, with 2016 consolidated sales of approximately $11 billion.

All Otsuka stories start by taking the road less travelled. Learn more about Otsuka in the U.S. at www.otsuka-us.com and connect with us on LinkedIn and Twitter at @OtsukaUS. Otsuka Pharmaceutical Co., Ltd.’s global website is accessible at www.otsuka.co.jp/en/.

Otsuka Pharmaceutical
(In Japan)
Jeffrey Gilbert
Leader, Pharmaceutical Public Relations
81-3-6361-7379
Gilbert.jeffrey@otsuka.jp

(In the U.S.)
Kimberly Whitefield                                      
Corporate Communications                                         
Otsuka America Pharmaceutical, Inc.
kimberly.whitefield@otsuka-us.com                         
+1-609-535-9259                               
_________________


[1] Lascano M., Poggio E. Kidney function assessment by creatinine-based estimation equations. Cleveland Clinic, Center For Continuing Education. 2010. [Accessed May 15, 2017]   http://www.clevelandclinicmeded.com/medicalpubs/diseasemanagement/nephrology/kidney-function/
[2] Torres VE, Harris PC et al. Tolvaptan in patients with autosomal dominant polycystic kidney disease. NEJM. 2012;367 (25): 2407-2418
[3] Thong KM, Ong ACM. The natural history of autosomal dominant polycystic kidney disease: 30-year experience from a single centre. QJM. 2013;2-8
[4] 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(7):1271-1276
[5] Rizk D, Jurkovitz C et al. Quality of life in Autosomal Dominant Polycystic Kidney Disease patients not yet on dialysis. Clin J Am Soc Nephrol. 2009;4:560-6
[6] Perlman RL, Finkelstein FO et al. Quality of life in chronic kidney disease (CKD): a cross-sectional analysis in the Renal Research Institute-CKD study. Am J Kidney Dis. 2005;45:658-66