From PharmiWeb, Press Release
OTSUKA’S JINARC® THE FIRST-EVER TREATMENT APPROVED IN EUROPE FOR ADULTS LIVING WITH ADPKD A CHRONIC GENETIC KIDNEY DISEASE
“Until now healthcare professionals have focused on treating the signs and symptoms of ADPKD with no specific treatment available to treat the disease” said Professor Ron T. Gansevoort University Medical Centre Groningen the Netherlands an expert in the field of polycystic kidney disease. “Tolvaptan represents a significant medical breakthrough in the management of ADPKD. For the first time healthcare professionals can modify the progression of the disease and preserve kidney function with the potential to improve patients’ quality of life and long-term outcomes.”
The marketing authorisation for tolvaptan is based on the findings of the pivotal Phase III randomised double-blind and placebo-controlled TEMPO 3:4 trial – the largest clinical study conducted in ADPKD to date.1 In the three-year study the rate of TKV increase over 3 years was significantly less for tolvaptan-treated subjects than for subjects receiving placebo: 2.80% per year vs 5.51% per year respectively (ratio of geometric mean 0.974; 95% CI 0.969 to 0.980; p <0.0001); these data demonstrate an approximate 50% significant reduction in the annual increase in TKV versus placebo.3 Furthermore tolvaptan showed a statistically significant reduction in the risk of multiple events of worsening kidney function kidney pain hypertension or albuminuria (hazard ratio=0.87 95% CI: 0.78-0.97 p=0.0095).3 The result of the key secondary composite endpoint is primarily attributed to effects on worsening kidney function (61.4% less likely with tolvaptan than with placebo) and medically significant kidney pain (35.8% less likely in tolvaptan-treated patients).3
Other than side effects associated with the mechanism of action of tolvaptan (eg thirst polyuria polliakuria) most side effects observed in ADPKD patients administered tolvaptan were comparable with those administered placebo.1 However a risk of liver injury was identified in patients with ADPKD taking tolvaptan. Elevation of alanine transaminase (ALT) was observed in 4.4% of patients on tolvaptan and 1.0% of patients on placebo.3 Two (2/957 0.2%) tolvaptan treated-patients as well as a third patient from an extension open label trial exhibited clinically significant increases in ALT with concomitant elevations in total bilirubin (BT).3 While these concomitant elevations were reversible with prompt discontinuation of tolvaptan they represent a potential for significant liver injury and patients taking tolvaptan will have to undergo monthly blood tests for the first 18 months of treatment with tolvaptan and three-monthly thereafter to mitigate this risk.3 Tolvaptan treatment must be initiated and monitored under the supervision of physicians with expertise in managing ADPKD and a full understanding of the risks of tolvaptan therapy including hepatic toxicity and monitoring requirements.3
“The progressive and hereditary nature of ADPKD is a physical and emotional burden on those living with the condition as well as their families and loved ones” said Tess Harris President of PKD International. “This approval is welcomed by the ADPKD community as it represents a step forward for the thousands of patients and carers throughout Europe who are affected by the disease.”
ADPKD is the most common inherited kidney disease primarily characterised by the proliferation and growth of multiple fluid-filled cysts in the kidney.16 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 (RRT) in the form of dialysis or a kidney transplant by the age of 54.27 ADPKD is the fourth leading cause of ESRD in adults8 and accounts for around 10% of patients requiring RRT.9
“It is a great honour to deliver the first treatment for ADPKD in Europe” said Tatsuo Higuchi President and Representative Director of Otsuka Pharmaceutical Co. Ltd.. “This approval is testament to the invaluable endeavours of the researchers and patients involved in the discovery and development of tolvaptan.”
Tolvaptan was first approved for patients with ADPKD in Japan in March 2014 and was approved for ADPKD in Canada in February 2015. Following this European marketing authorisation Otsuka will continue to work with local authorities in countries throughout Europe to help ensure that eligible ADPKD patients are able to access tolvaptan.
About JINARC® (tolvaptan) in ADPKD
Tolvaptan is a potent vasopressin V2 receptor antagonist that has been proven to slow cyst growth and renal function decline in patients with ADPKD in CKD stage one to three with evidence of rapidly progressive disease.1
The formation of cysts associated with ADPKD can lead to significant kidney damage even when kidney function is not affected10 and cause complications that include chronic and acute pain hypertension and kidney failure.1 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. 11-13
By selectively blocking vasopressin from its V2 receptor tolvaptan decreases cyst cell proliferation and fluid secretion ultimately reducing cyst development and renal clinical events associated with disease progression.14 [Read more]
Funding the Cure
KANSAS CITY, Mo. - While he didn't win the grand prize in the Food Network's cooking competition, Chopped All-Stars, New York City Chef Michael Psilakis still brought a ton of awareness to a Kansas City charity. He rallied for the Polycystic Kidney Disease Foundation throught the fourth season of the show.
Psilakis' dad died from complications related to polycystic kidney disease. The disease causes cysts to grow on a person's kidneys. A normal kidney is the size of an adult fist, but the kidney's of people living with the disease can blow up to the size of a football and weigh up to 30 pounds each.
If Psilakis wins, he'll donate $75,000 cash prize to the Polycystic Kidney Disease Foundation, a local nonprofit organization.
To learn more about the disease, including the warning sighs you should look out for, head over to Polycystic Kidney Disease Foundation website.
Seeking Life
Kevin Heuerman, a Sedalia husband and father, has polycystic kidney disease. He said his biggest wish is to get a call to come to Research Medical Center in Kansas City for a kidney transplant.
“A healthy kidney is about the size of a fist,” Heuerman said. “Mine, right now, they say are the size of footballs.”
The disease has covered Heuerman’s kidneys with growing cysts, causing the organs to lose function. He spends 12 hours each week at a clinic for dialysis, time that keeps him away from his family. He said it’s been tough on his son.
“He’ll be 10 in July,” Heuerman said. “It’s hard to tell him sometimes that I have to rest or I can’t go out and do this or that.”
With his kidneys functioning at less than 10 percent, Heuerman has been placed on the donor registry. The average wait is three to five years, so his wife has gone in search of that kidney herself.
“We’re best friends and there’s nothing I wouldn’t do for him,” said Melissa Heuerman.
Her first stop was Facebook, where a page calling attention to the family’s need gained traction overnight.
“We’ve had people share it in California, Hawaii and Arkansas,” she said.
She also has a sign she takes with her everywhere she goes.
“Impact Signs and Lighting called us and said that they would like to put it on my car for free,” Melissa Heuerman said.
“I knew when it was going to happen and we started doing all the testing – I knew that she’s not the kind of person that can be quiet about anything,” Kevin Heuerman said. [Read more]
Giving Life
STRANGE as it may sound, the nonprofit dialysis organization I lead works very hard to help patients leave dialysis, or avoid it entirely. Instead, we help them get a transplant.
For many people with chronic kidney failure, a kidney transplant offers the best chance at the lifestyle they enjoyed before diagnosis. But until quite recently, nature often thwarted good intentions because of tissue mismatches. Seattle Times reporter JoNel Aleccia recently reported a very hopeful story about technology to arrange kidney swaps, matching willing donors with compatible recipients, greatly extending the possibilities for transplants.
Without a transplant, people with kidney failure must go on dialysis three days a week or daily for life, relying on a machine to do the waste-removal work of a kidney.
Transplant recipients often live longer, feel better and have more independence than people on dialysis. So we actively encourage our patients to seek a transplant. More than a quarter of Northwest Kidney Centers’ dialysis patients now qualify for the waitlist to get a kidney.
We work with each individual to clear barriers to transplant. Some receive free treatment from volunteer dentists who make sure oral infection doesn’t bump them off the list. We coach patients to speak out about their need for a kidney wherever they go, in hopes of appealing to a prospective living donor.
Last year, 87 of our patients received the gift of a new kidney. A kidney patient here is almost twice as likely to get a transplant as in other parts of the country. That’s a testament to hard work by our doctors and staff to make transplants a priority and to support patient enthusiasm to get a new kidney. Seattle is fortunate to have four excellent transplant centers — Swedish Medical Center, the University of Washington Medical Center, Virginia Mason Hospital and Medical Center, and Seattle Children’s Hospital.
While many of our waiting patients have a friend or relative ready and willing to give a kidney, others aren’t as lucky. That’s where you come in.
Be sure your driver’s license has a red heart indicating you’ll be an organ donor after you die. Take care of that task when you renew your license, or better still, go now to www.donatelifenw.org and sign up.
Open your mind to the possibility of becoming an organ donor while you live by giving one of your two kidneys. If you don’t already know someone who needs it, chances are you will hear of a friend (or a friend of a friend) in need. Your recipient’s insurance would cover the cost of your surgery and you would need a few weeks off to recover. [Read more]
