Wednesday, November 30, 2011

Over 900 Biotechnology Medicines in Development, Targeting More than 100 Diseases

Over 900 Biotechnology Medicines in Development, Targeting More than 100 Diseases
September 14, 2011

Biotechnology has opened the door to the discovery and development of new types of human therapeutics. Advancements in both cellular and molecular biology have allowed scientists to identify and develop a host of new products. These cutting-edge medicines provide significant clinical benefits, and in many cases, address therapeutic categories where no effective treatment previously existed.

Innovative, targeted therapies offer enormous potential to address unmet medical needs of patients with cancer, HIV/AIDS, and many other serious diseases. These medicines also hold the potential to help us meet the challenge of rising healthcare costs by avoiding treatment complications and making sure each patient gets the most effective care possible.

Approved biotechnology medicines already treat or help prevent heart attacks, stroke, multiple sclerosis, leukemia, hepatitis, congestive heart failure, lymphoma, kidney cancer, cystic fibrosis, and other diseases. These medicines use many different approaches to treat disease as do medicines currently in the pipeline.

America's biopharmaceutical research companies have 901 biotechnology medicines and vaccines in development to target more than 100 debilitating and life- threatening diseases, such as cancer, arthritis and diabetes, according to a new report [] by the Pharmaceutical Research and Manufacturers of America (PhRMA). The medicines in development—all in either clinical trials or under Food and Drug Administration review—include 353 for cancer and related conditions, 187 for infectious diseases, 69 for autoimmune diseases and 59 for cardiovascular diseases.

The biotechnology medicines now in development make use of these and other state-of- the-art approaches. For example:

•A genetically-modified virus-based vaccine to treat melanoma.
•A monoclonal antibody for the treatment of cancer and asthma.
•An antisense medicine for the treatment of cancer.
•A recombinant fusion protein to treat age-related macular degeneration.


Autoimmune Diseases: Autoimmunity is the underlying cause of more than 100 serious, chronic illnesses, targeting women 75 percent of the time. Autoimmune diseases have been cited in the top 10 leading causes of all deaths among U.S. women age 65 and younger, representing the fourth largest cause of disability among women in the United States.

Blood Disorders: Hemophilia affects 1 in 5,000 male births. About 400 babies are born with hemophilia each year. Currently, the number of people with hemophilia in the United States is estimated to be about 20,000, based on expected births and deaths since 1994.

Sickle cell disease is an inherited disease that affects more than 80,000 people in the United States, 98 percent of whom are of African descent.

Von Willebrand disease, the most common inherited bleeding condition, affects males and females about equally and is present in up to 1 percent of the U.S. population.

Cancer: Cancer is the second leading cause of death by disease in the United States—1 of every 4 deaths—exceeded only by heart disease. This year nearly 1.6 million new cancer cases will be diagnosed, 78 percent of which will be for individuals ages 55 and older.

Cardiovascular Diseases (CVD): CVD claims more lives each year than cancer, chronic lower respiratory diseases, and accidents combined. More than 82 million American adults—greater than one in three—had one or more types of CVD. Of that total, 40.4 million were estimated to be age 60 and older.

Diabetes: In the United States, 25.8 million people, or 8.3 percent of the population, have diabetes. An estimated 18.8 million have been diagnosed, but 7 million people are not aware that they have the disease. Another 79 million have pre-diabetes. Diabetes is the seventh leading cause of death in the United States.

Genetic Disorders: There are more than 6,000 known genetic disorders. Approximately 4 million babies are born each year, and about 3 percent-4 percent will be born with a genetic disease or major birth defect. More than 20 percent of infant deaths are caused by birth defects or genetic conditions (e.g., congenital heart defects, abnormalities of the nervous system, or chromosomal abnormalities).

Alzheimer’s Disease: In 2010 there were an estimated 454,000 new cases of Alzheimer’s disease. In 2008, Alzheimer’s was reported as the underlying cause of death for 82,476 people. Almost two-thirds of all Americans living with Alzheimer’s are women.

Parkinson's Disease: This disease has been reported to affect approximately 1 percent of Americans over age 50, but unrecognized early symptoms of the disease may be present in as many as 10 percent of those over age 60. Parkinson's disease is more prevalent in men than in women by a ratio of three to two.

Asthma: An estimated 39.9 million Americans have been diagnosed with asthma by a health professional within their lifetime. Females traditionally have consistently higher rates of asthma than males. African Americans are also more likely to be diagnosed with asthma over their lifetime.

Skin Diseases: More than 100 million Americans—one-third of the U.S. population—are afflicted with skin diseases.