Breaking News
October 16, 2018 - New blood test for pediatric brain tumor patients offers safer approach than surgical biopsies
October 16, 2018 - Age-related estrogen increase may be the culprit behind inguinal hernias in men
October 16, 2018 - Skills-Based Intervention Did Not Cut Systolic BP After Stroke, TIA
October 16, 2018 - Researchers uncover new role of TIP60 protein in controlling tumour formation
October 16, 2018 - Behind the scenes of a lifesaving heart surgery
October 16, 2018 - ‘To See the Suffering’
October 16, 2018 - Drinking concentrated rosemary extract can boost memory by up to 15%, shows research
October 16, 2018 - Medicare Advantage riding high as new insurers flock to sell to seniors
October 16, 2018 - NHS tackles prescription fraud to save millions
October 16, 2018 - New molecular switch may help develop sophisticated photomedications
October 16, 2018 - Health Highlights: Oct. 12, 2018
October 16, 2018 - Study holds promise for new pediatric brain tumor treatment
October 16, 2018 - Patient advocate uses MRI scans to create art and spark conversations about life with illness
October 16, 2018 - Fish oil based diets may suppress growth and spread of breast cancer cells
October 16, 2018 - Number of VHA facilities offering acupuncture has increased rapidly
October 16, 2018 - Influential Leapfrog Group jumps in to rate 5,600 surgery centers
October 16, 2018 - HIV-infected infants more likely to acquire congenital cytomegalovirus infection
October 16, 2018 - Study pinpoints new marker that can predict Crohn’s disease subtype
October 16, 2018 - Simple procedure could be efficacious intervention for failed back surgery
October 16, 2018 - New research identifies modifiable dementia risk factor in elderly people
October 16, 2018 - Zebrafish study uncovers molecular ‘brake’ that helps control eye lens development
October 16, 2018 - Overlapping copy number variations underlie autism and schizophrenia in Japanese patients
October 16, 2018 - Majority of Americans’ ancestry can be traced through existing DNA databases
October 16, 2018 - Patients coerced into mental health care less likely to perceive treatment as effective
October 16, 2018 - Healthy elders can consume walnuts without having negative impact on weight gain, finds study
October 16, 2018 - Interactive robot helps older people exercise and detects underlying health problems
October 16, 2018 - What you need to know about autism spectrum disorder
October 16, 2018 - Antidepressants can be used to treat Alzheimer’s disease
October 16, 2018 - Study uncovers important role of PRMT1 in dilated cardiomyopathy
October 16, 2018 - Nutritional quality of breakfast linked to cardiovascular and metabolic risk factors in children
October 16, 2018 - Study uses novel approach to investigate genetic origins of mental illnesses
October 16, 2018 - Scientists develop dual anthrax-plague vaccine
October 16, 2018 - Poor Outcomes for Hispanic Infants With Congenital Heart Dz
October 16, 2018 - Global study finds youngest in class more likely to be diagnosed with ADHD
October 16, 2018 - Researchers sequence two selfish genes in the fungus Neurospora intermedia
October 16, 2018 - Survey results highlight the need for better communication between patients and HCPs about bacterial vaginosis
October 16, 2018 - Researchers develop fibrin-targeting immunotherapy to protect against neurodegeneration
October 16, 2018 - Researchers create open access database on healthy immunity
October 16, 2018 - Rice University chemist wins big award to study small surfaces
October 16, 2018 - Study finds 43% drop in stroke rate
October 16, 2018 - Researchers identify basic relationships of cell cycle and cellular senescence in the placenta
October 16, 2018 - UA professor receives NSF grant to develop antifouling materials for medical implants
October 16, 2018 - Obesity Doubles Odds for Colon Cancer in Younger Women
October 16, 2018 - Adults with ADHD not constrained in creativity
October 16, 2018 - Raising visibility for people and students with chronic illness and disability
October 16, 2018 - Allele awarded NIH grant to develop nanoantibody therapies for treatment of sepsis
October 16, 2018 - Only 59% of young adults undergoing surgery are fluid responsive
October 16, 2018 - Research points to potential new treatment for hearing loss
October 16, 2018 - MDI Biological Laboratory receives $1.2 million SEPA grant to promote data literacy
October 16, 2018 - Vast majority of dementia cases may arise from spontaneous genetic errors
October 16, 2018 - New project aims to deliver fast, effective treatment for autoimmune rheumatic diseases
October 16, 2018 - Study identifies molecular switch that controls fate of milk-producing breast cells
October 16, 2018 - Research shows diet has little influence on precursor to gout
October 16, 2018 - “Without Dr. Shumway doing his miracle work, three generations would not be here”: A Stanford heart transplant patient’s story
October 16, 2018 - Non-invasive brain stimulation sheds light on neurobiology underlying implicit bias
October 16, 2018 - Researchers demonstrate integrated technique to control production of cell therapeutics
October 16, 2018 - Breast tomosynthesis detects 34% more tumors than traditional mammography
October 16, 2018 - Rhode Island Hospital, Brown receive $800,000 grant to keep up fight against opioid epidemic
October 16, 2018 - UVA partners with health systems in AVIA network’s Medicaid Transformation Project
October 16, 2018 - Trevena Announces Oliceridine FDA Advisory Committee Meeting Outcome
October 16, 2018 - Study reveals early warning signs of heart problems in patients with newly diagnosed lupus
October 16, 2018 - Connecting the dots of Alzheimer’s disease
October 16, 2018 - New publication offers evidence-based content for global breast imaging medical community
October 16, 2018 - ‘EinsteinVision’ that improves hand-eye coordination of surgeons introduced at Harefield Hospital
October 16, 2018 - WRAIR clinical study evaluates safety and immunogenicity of Marburg vaccine
October 16, 2018 - Ketamine can be considered as alternative to opioids for short-term pain control in ED
October 16, 2018 - Endurance exercise training beneficially alters gut microbiota composition
October 15, 2018 - FDA Approves Yutiq (fluocinolone acetonide intravitreal implant) for Chronic Non-Infectious Posterior Segment Uveitis
October 15, 2018 - Birthing Options for Full-Term Pregnancy
October 15, 2018 - Stressed, toxic, zombie cells seen for first time in Alzheimer’s
October 15, 2018 - Concussion researchers study head motion in high school football hits | News Center
October 15, 2018 - Neuropsychiatric symptoms related to earliest stages of Alzheimer’s brain pathology
October 15, 2018 - Neck collar device may help protect the brain of female high school soccer players
October 15, 2018 - Research reveals how the inner ear processes speech
October 15, 2018 - Many parents still skeptical about safety and effectiveness of flu shot, survey finds
October 15, 2018 - Payer Policies May Discourage Non-Pharma Tx for Low Back Pain
October 15, 2018 - Exercise may delay cognitive decline in people with rare Alzheimer’s disease
October 15, 2018 - Researchers modify CRISPR to reorganize genome | News Center
October 15, 2018 - Innovative brain tumor operation set to tailor to patients’ needs
October 15, 2018 - Findings offer new insight into early changes that occur during AD pathology
FDA Approves Jynarque (tolvaptan) to Slow Kidney Function Decline in Rapidly Progressing Autosomal Dominant Polycystic Kidney Disease

FDA Approves Jynarque (tolvaptan) to Slow Kidney Function Decline in Rapidly Progressing Autosomal Dominant Polycystic Kidney Disease

image_pdfDownload PDFimage_print

FDA Approves Jynarque (tolvaptan) to Slow Kidney Function Decline in Rapidly Progressing Autosomal Dominant Polycystic Kidney Disease

April 24, 2018 — TOKYO–(BUSINESS WIRE)–Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces that the U.S. Food and Drug Administration (FDA) has approved Jynarque (tolvaptan) as the first drug treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD).

ADPKD is a genetic disease with consequences that can lead to dialysis or kidney transplantation. It is a progressively debilitating and often painful disorder in which fluid-filled cysts develop in the kidneys over time. These cysts enlarge the kidneys and impair their ability to function normally, leading to kidney failure in most patients.3 ADPKD is diagnosed in approximately 140,000 people in the U.S.,4,5 and impacts families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.6,7

The efficacy of tolvaptan was demonstrated in two pivotal trials, lasting one year and three years, respectively. In the one-year REPRISE study, the primary endpoint was the treatment difference in the change of eGFR from pretreatment baseline to post-treatment follow-up, annualized by dividing by each subject’s treatment duration. In the randomized period, the change of eGFR from pretreatment baseline to post-treatment follow-up was −2.3 mL/min/1.73 m2/year with tolvaptan as compared with −3.6 mL/min/1.73 m2/year with placebo, corresponding to a treatment effect of 1.3 mL/min/1.73 m2/year (p

The primary endpoint in TEMPO 3:4 study was the intergroup difference for rate of change of total kidney volume (TKV) normalized as a percentage. The trial met its pre-specified primary endpoint of 3-year change in TKV (p

Jynarque can cause serious and potentially fatal liver injury, and acute liver failure requiring liver transplantation has been reported. Jynarque has been associated with elevations of blood alanine and aspartate aminotransferases (ALT and AST), with infrequent cases of concomitant elevations in bilirubin-total (BT). To ensure the safety of patients taking Jynarque, it is necessary to measure ALT, AST and bilirubin before initiating treatment, at 2 weeks and 4 weeks after initiation, then monthly for 18 months and every 3 months thereafter, for as long as the patient is on Jynarque (tolvaptan) treatment. Because of the risks of serious liver injury, Jynarque is available only through a restricted distribution program supported by a Risk Evaluation and Mitigation Strategy (REMS) Program approved by the FDA. For more information about Jynarque, please visit www.JYNARQUE.com.

“The progressive nature of ADPKD means that kidney function gets worse over time, eventually leading to end-stage renal disease. This progression happens more rapidly for some patients than others.” said Michal Mrug, M.D., Associate Professor at the University of Alabama at Birmingham and investigator on the REPRISE trial. “Today’s approval is great news for adults at risk of rapidly progressing ADPKD because by slowing the decline in kidney function, this therapy may give them more time before kidney transplant or dialysis.”

Andy Betts, CEO of the PKD Foundation, observed, “Today is an historic day in providing hope to patients with autosomal dominant polycystic kidney disease, and we are thrilled to be a part of this first milestone in treatment. For the past 35 years, our goal has been to walk with PKD patients every step of the way. It is gratifying to play a part in the inception of the discovery of this treatment, and to see it come to fruition. We hope that this is just the beginning of a new chapter for adults at risk of rapidly progressing ADPKD who suffer from the disease.”

Also, Tatsuo Higuchi, president and representative director of Otsuka Pharmaceutical Co., Ltd., commented, “This approval is important news for many adults at risk of rapidly progressing ADPKD in the U.S., who have had no therapeutic alternatives to delay the eventual end-stage interventions of dialysis or kidney transplantation. We are humbled to be able to offer an earlier, proactive course of action to slow the progression of this disease, which we know means so much to patients, their families and healthcare providers. Simultaneously, we are grateful to the patients and researchers who through their continued commitment made this milestone possible.”

About ADPKD

ADPKD is a progressively debilitating and often painful genetic disorder in which fluid-filled cysts develop in the kidneys over time. These cysts enlarge the kidneys and impair their ability to function normally, leading to kidney failure in most patients. ADPKD can impact quality of life, and is also associated with cardiovascular complications that can cause death.3 ADPKD is diagnosed in approximately 140,000 people in the U.S.,4,5 and is the fourth leading cause of end-stage renal disease.1,2

ADPKD impacts families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.6,7 Risk factors for rapid disease progression include having a greater TKV than expected for age,8,9 family history of end-stage renal disease before 58 years of age,10 high blood pressure before 35 years of age,11 certain urologic events before 35 years of age,12 a historical decline in eGFR of ≥5 mL/min/1.73 m2 within 1 year,10 certain inherited genetic profiles,12 or male sex.12

About Jynarque (tolvaptan)

Jynarque is a selective vasopressin V2-receptor antagonist indicated to slow kidney function decline in adults at risk of rapidly progressing ADPKD. The medication has been approved as a treatment for adults with ADPKD in Japan, the EU, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey and Taiwan. See local prescribing information for specific indications in each country.

The FDA approval of Jynarque is supported by data from the TEMPO 3:4 (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes) and REPRISE (Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD) clinical trials published in the New England Journal of Medicine in November 2012 and November 2017, respectively.

Jynarque will be sold in a 28-day treatment pack at a wholesale acquisition cost of $13,041.10.

More information for U.S. Healthcare Providers can be found at www.JYNARQUEhcp.com.

Otsuka, maker of Jynarque (tolvaptan), is committed to helping patients access the medications they need.

Otsuka is committed to helping adults at risk of rapidly progressing ADPKD and will work closely with these patients, providers and payers to ensure Jynarque is broadly accessible. Otsuka offers a number of support programs for eligible patients, including the Jynarque Copay Savings program, which ensures that eligible commercially insured patients do not pay more than $10 per prescription for Jynarque. Further details about program limitations and legally required exclusions are available at www.JYNARQUE.com/Copay-Savings. The Otsuka Patient Assistance Foundation (OPAF), a separate and independent entity, provides support for eligible patients who are unable to afford their medication. OPAF’s mission is to eliminate financial barriers to drug therapies for uninsured or underinsured patients. For more information, please visit www.otsukapatientassistance.com.

About Otsuka

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 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 R&D programs in areas including nephrology and cardiology. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does.

Otsuka Pharmaceutical Company is a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan. The Otsuka group of companies employed 46,000 people worldwide and had consolidated sales of approximately USD 11.1 billion in 2017.

All Otsuka stories start by taking the road less travelled. Learn more about Otsuka Pharmaceutical Company on its global website at https://www.otsuka.co.jp/en. Learn more about Otsuka in the U.S. at www.otsuka-us.com and connect with us on Twitter at @OtsukaUS.

1 United States Renal Data System. National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, 2016. Available at: https://www.usrds.org/2016/download/v2_ESRD_16.pdf (accessed August 8, 2017).

2 Lentine, K. L. (2015). Renal Function and Healthcare Costs in Patients with Polycystic Kidney Disease. Clinical Journal of the American Society of Nephrology, 5, 1471-1479.

3 Chebib F, Torres V et al. Autosomal dominant polycystic kidney disease: core curriculum 2016. Am J Kidney Dis. May 2016; 67(5): 792-810.

4 Data on file. Otsuka America Pharmaceutical, Inc., 2018

5 Willey, CJ et al. Epidemiology of Autosomal Dominant Polycystic Kidney Disease in the United States. Poster presented at American Society of Nephrology Kidney Week; 2013 Nov 5-10; Atlanta, GA

6 Autosomal Dominant Polycystic Kidney Disease Fact Sheet. NIH Reports. Accessed online on March 30 2018 at https://report.nih.gov/NIHfactsheets/ViewFactSheet.aspx?csid=29&key=A#A

7 Torres VE, Grantham JJ. Cystic diseases of the kidney. In: Taal MW, Chertow GM, Mardsen PA, Skorecki K, Yu ASL, Brenner BM, eds. Brenner & Rector’s The Kidney.Philadelphia, PA: Elsevier Saunders; 2012:1626-1667

8 Irazabal MV. Imaging Classification of autosomal dominant polycystic kidney disease: a simple model for selecting patients for clinical trials. J Am Soc Neph. 2015 vol. 26 no. 1 160-172.

9 Chapman A. et al. Kidney Volume and Functional Outcomes in Autosomal Dominant Polycystic Kidney Disease. Clin J Am Soc Nephrol 2012; 7; 479-486.

10 Gansevoort RT. Recommendations for the use of tolvaptan in autosomal dominant polycystic kidney disease: a position statement on behalf of the ERA-EDTA Working Groups on Inherited Kidney Disorders and European Renal Best Practice. Nephrology Dialysis Transplantation 2016, Vol. 31, 337–3489

11 Schrier JASN 2010/p891/col1/1/ln1-3]

12 Cornec Le Gall E. The PROPKD score: a new algorithm to predict renal survival in autosomal dominant polycystic kidney disease. J Am Soc Nephrol. 2016 Mar;27(3):942-51

Source: Otsuka Pharmaceutical Co., Ltd.

Posted: April 2018

Jynarque (tolvaptan) FDA Approval History

Tagged with:

About author

Related Articles