The Need

Therapeutic areas

Chronic progressive diseases such as congestive heart failure (CHF) and type 2 diabetes afflict hundreds of millions of people worldwide, and the incidence is growing rapidly. At present there are no definitive treatments for either disease that can fully restore a person’s health. Obesity and non-alcoholic fatty liver disease (NAFLD) are also two growing threats to global health, and pulmonary hypertension remains a rare yet burdensome and fatal disease. All of these debilitating chronic diseases pose an unsustainable burden on the lives of patients and their families, on medical practitioners, and on economies crippled by health care costs.

    • Congestive heart failure

      Congestive heart failure (CHF) is a chronic disease characterized by the inability of the heart to pump sufficient blood to meet the body’s demands. It is a progressive and fatal chronic disease, and symptoms worsen over time. CHF can be caused by many conditions that damage the heart muscle, including heart attacks, infections, alcohol or drug abuse as well as high blood pressure, valve disease, thyroid disease, kidney disease, diabetes, or heart defects present at birth. People with severe CHF may need a mechanical heart pump or a heart transplant.

      CHF afflicts approximately 6 million people in the United States and 28 million people globally.1 Projections show that the prevalence of heart failure will increase 46 percent by 2030.1 In 2012, the annual cost of heart failure in the United States was $31 billion, with projections suggesting that increasing prevalence will increase medical costs to $70 billion in 2030.1

    • Acute decompensated heart failure

      Acute Decompensated Heart Failure (ADHF) is a severe episode of acute respiratory distress in which a CHF patient experiences a sudden or gradual onset of the signs of heart failure that requires either initiation or escalation of treatment to relieve symptoms and prevent death. ADHF is caused by severe fluid congestion of the body’s organs, especially the lungs, due to the failing heart providing inadequate circulation.

      Symptoms of ADHF can include difficulty breathing, waking up from sleep gasping for air, fatigue, swelling of the legs or feet, as well as chest pain and pressure. Most episodes require immediate emergency room visits or hospitalizations for treatment.

      Current treatment options for ADHF primarily involve reducing the organ fluid levels with diuretics, delivered intravenously, and stabilizing heart function with vasodilators such as nitroglycerin. Additional treatments can include providing continuous air pressure using a face mask, as well as an ultrafiltration process to remove fluids in people with ADHF associated with kidney failure.

      In the United States, ADHF accounts for approximately half of all CHF-related causes of hospitalization.2 From 2005-2010, the average estimated number of ADHF hospitalizations in the U.S. was a staggering 1.76 million.3 Annual mortality following an ADHF hospitalization is about 30%.4

    • Type 2 diabetes

      Type 2 diabetes is by far the most common form of diabetes, accounting for approximately 90% of the world’s diabetes cases. It renders the body unable to regulate blood sugar levels, resulting in a range of problems, including heart disease, kidney failure, blindness, and the loss of toes and fingers to nerve damage.

      Genetics and lifestyle both play roles in causing type 2 diabetes, and the risk of having the disease increases as one gets older. This chronic condition has been linked to obesity, genetic risk factors and inactivity. Managing type 2 diabetes varies among patients but often includes medication and insulin therapy. Currently, there is no known way to cure it.

      The incidence of diabetes is staggering, with 422 million adults around the world living with this debilitating disease, a number that has nearly quadrupled from 108 million in 1980.5 In 2012, 1.5 million deaths were directly caused by diabetes. By 2030, the World Health Organization (WHO) projects diabetes will be the seventh-leading cause of death in the world.6

      The costs are overwhelming: The direct global annual cost of diabetes is $827 billion. In the United States, people with diagnosed diabetes incur average medical expenditures of about $14,999 per year.7

    • Pulmonary hypertension

      Pulmonary hypertension (PH) is a rare disease that occurs when arteries within the lungs become narrowed, making blood flow more difficult. The resulting blockage within these arteries – known as pulmonary arteries – creates abnormally high blood pressure that strains the heart’s right ventricle, causing it to expand and eventually weaken, often to the point of failure. Sufferers of this progressively worsening and fatal disease experience a variety of symptoms, including shortness of breath, fatigue, dizziness, fainting spells, chest pressure and pain, swelling in the ankles, abdomen and legs, bluish lips and skin, an increased pulse and heart palpitations. Resulting complications from PH include right-sided heart failure, blood clots, irregular heartbeats and bleeding in the lungs, all of which can result in death.

      The causes of pulmonary hypertension vary widely. Primary risk factors include genetics – PH is more likely if two or more family members have it – as well as chronic liver disease, rheumatic disorders, blood clots in the lungs, congenital heart defects, chronic obstructive pulmonary diseases, connective tissue disorders, sleep apnea,  living at high altitudes, and use of stimulants such as methamphetamines and the diet drug “fen-phen.”

      Currently, the only cure for pulmonary hypertension is lung transplantation; a shortage of donors keeps this from being a viable solution for many patients. More common treatments – oral, inhaled, intravenous and subcutaneous – are primarily focused on alleviating disease symptoms or causes.

      Pulmonary hypertension is estimated to affect approximately 100,000 people worldwide. In Europe, primary pulmonary hypertension is responsible for approximately 200 deaths per year and has an incidence rate of approximately three cases per million per year.8 In the US an estimated 300 new cases of primary pulmonary hypertension are diagnosed each year, according to the American Lung Association.

    • Obesity

      Obesity is the accumulation of an excessive amount of fat in the body. This increasingly more common condition is a major risk factor for several serious diseases, including heart disease and stroke, type 2 diabetes, musculoskeletal disorders such as osteoarthritis, as well as endometrial, breast and colon cancer. The World Health Organization (WHO) defines the fundamental cause of obesity as an energy imbalance between calories consumed and calories expended. It is measured by a body mass index (BMI) – weight-to-height ratio – greater than or equal to 30.

      The numbers are overwhelming: In 2014 more than 600 million adults were obese. That is about 13% of the world’s adult population – 11% of men and 15% of women. And it’s on the rise around the world: The global prevalence of obesity has more than doubled since 1980.9

      Along with being a primary risk factor for many serious chronic diseases, the medical cost of obesity is also staggering. In 2008, the estimated annual medical cost of obesity in the United States was $147 billion. The annual medical costs for people who are obese were $1,429 higher than those of normal weight.10

      Non-alcoholic fatty liver disease

      Non-alcoholic fatty liver disease (NAFLD) is the build-up of extra fat in liver cells not caused by alcohol. It occurs when liver tissue cannot properly break down fat. This may cause the liver to swell, which, over time, can result in scarring – known as cirrhosis – and lead to liver cancer or liver failure. The more severe form of NAFLD is called non-alcoholic steatohepatitis (NASH), one of the leading causes of cirrhosis in adults in the United States.

      While NAFLD often has no symptoms, some symptoms may include the following: fatigue, weakness, weight loss, loss of appetite, nausea, abdominal pain, spider-like blood vessels, yellowing of the skin and eyes, itching, fluid build up and swelling of the legs and abdomen, and mental confusion.

      NAFLD tends to develop in people who are overweight or obese, or have lost weight rapidly. Type 2 diabetes, high cholesterol, high levels of triglycerides in the blood and sleep apnea are some of the main risk factors for developing NAFLD. However, some people develop NAFLD even if they do not have any of these risk factors.

      One-third of the US population is estimated to have NAFLD.11 Prevalence in Asia and Europe is approximately 25%.12 Currently, there is no standard treatment for NAFLD. Physicians instead focus on treating the risk factors that contribute to the liver disease.13

1Mozaffarian D, Benjamin EJ, Go AS, et al. Heart disease and stroke statistics-2015 update: a report from the American Heart Association. Circulation. 2015;131(4):e29-e322.
2Chang PP, Chambless LE, Shahar E, et al. Incidence and Survival of Hospitalized Acute Decompensated Heart Failure in Four US Communities (from the Atherosclerosis Risk in Communities Study). The American journal of cardiology. 2014;113(3):504-510. doi:10.1016/j.amjcard.2013.10.032.
3Agarwal SK, Wruck L, Quibrera M, et al. Temporal Trends in Hospitalization for Acute Decompensated Heart Failure in the United States, 1998–201. Am. J. Epidemiol. 2016; 183(5):462-470.
4Dhingra A, Garg A, Kaur S, et al. Epidemiology of heart failure with preserved ejection fraction. Curr Heart Fail Rep. 2014; 11(4):354–365.
5Worldwide trends in diabetes since 1980: a pooled analysis of 751 population-based studies with 4·4 million participants. The Lancet, Volume 387, Issue 10027, 1513 – 1530.
6http://www.who.int/mediacentre/factsheets/fs312/en/
7http://www.healthcostinstitute.org/files/HCCI%20Diabetes%20Issue%20Brief%205-7-15.pdf
8http://www.efpia.eu/diseases/47/59/Pulmonary-Hypertension
9http://www.who.int/mediacentre/factsheets/fs311/en/
10http://content.healthaffairs.org/content/28/5/w822.full.pdf+html
11http://www.clevelandclinicmeded.com/medicalpubs/diseasemanagement/hepatology/nonalcoholic-fatty-liver-disease/Default.htm
12http://link.springer.com/article/10.1007%2Fs10620-015-3728-3
13http://www.mayoclinic.org/diseases-conditions/nonalcoholic-fatty-liver-disease/basics/risk-factors/con-20027761

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