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Fighting for Children's Health

America’s biopharmaceutical companies are researching 316 medicines to help meet the unique health care needs of children.

Medicine has played a key role in the fight against diseases that impact children. Infant mortality has sunk to record lows, and new vaccines protect children against many childhood diseases. 

A child born today can expect to live 30 years longer than a child born a century ago, thanks in part to major advances in treatment. Biopharmaceutical research companies are working to continue this progress and to meet new health challenges specific to children. Infant mortality has sunk to record lows. New vaccines protect children against many childhood diseases. Antibiotics prevent deaths from pneumonia and other infectious diseases that used to claim the lives of children. America’s biopharmaceutical

companies are researching 316 medicines to help meet the unique health care needs of children and adolescents. In addition to creating new medicines specifically for children, biopharmaceutical research companies are testing many existing medicines to determine safe and effective dosage levels for children. Considerable progress in the fight against diseases that impact children has been achieved in recent decades, with medicines playing a key role. 

1 in 20 Afflicted by ADHD

Attention-deficit/hyperactivity disorder (ADHD) is one of the most common reasons children are referred for mental health services. It affects as many as one in every 20 children. Although most children with ADHD have normal or above-normal intelligence, 40 to 60 percent have serious learning difficulties.

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1 out of Every 110 Children Today are Diagnosed with Autism

Autism is more prevalent than childhood cancers, multiple sclerosis, and cystic fibrosis combined. The median age of earliest ASD diagnosis is between 4.5 and 5.5 years, but for 51 percent–91 percent of children with an ASD, developmental concerns had been recorded before the age of three. 

Juvenile Rheumatoid Arthritis Affects 300,000 Children


Thanks in part to major treatment advances, 82% of children diagnosed with cancer will survive five years or longer today, compared to 58% three decades ago, according to the American Cancer Society. 

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Every 3 Seconds

A Child Dies – In Most Cases from an Infectious Disease

Infectious diseases are now the world’s biggest killer of children and young adults, killing 13 million per year. More widespread use of low-cost vaccines could prevent 1.6 million deaths a year among children under the age of five. Yet today, one in five children is still not fully immunized against major killer diseases.

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Medicines in the Pipeline

Biopharmaceutical research companies are testing 316 medicines to meet the special health needs of infants, children and adolescents. These medicines offer hope that the significant improvements achieved in children’s health over the past few decades will continue and even accelerate.

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Related Medicines

  • Anti-IL-5

    Asthma is one of the most common chronic disorders in childhood and affects an estimated 7.1 million American children under the age of 18, according to the American Lung Association. Eosinophils (a type of white blood cell) are important for killing parasites in the body, but in asthma they can accumulate in lung tissue and cause damage to the lining of the air passages. Interleukin-5 (IL-5) is essential for the production, activation and maturation of eosinophils. A monoclonal antibody in development is an anti-IL-5 that inhibits IL-5 resulting in a sustained reduction in the numbers of eosinophils accumulating in the lungs and stopping those already there from causing damage.

  • Human-derived antibody that binds to TNF alpha

    Crohn’s disease, a chronic disorder that causes inflammation of the digestive or gastrointestinal tract, affects about 140,000 American children under the age of 18, according to the Crohn’s and Colitis Foundation of America. One medicine already approved to reduce the signs and symptoms of moderate to severe Crohn’s disease in adults, and now in pediatric trials, is a human-derived antibody that binds to human tumor necrosis factor alpha (TNF alpha). TNF alpha is a protein responsible for the inflammatory process in the intestines that is caused by Crohn's disease.

  • DPP-4 (dipeptidyl peptidase-4) inhibitor

    Diabetes affects about 215,000 Americans under the age of 20, according to the American Diabetes Association. One medicine, already approved for use in adults, is being tested in children with type 2 diabetes. The medicine was the first DPP-4 (dipeptidyl peptidase-4) inhibitor approved in the United States for the treatment of type 2 diabetes. The medicine enhances a natural body system called the incretin system, which helps to regulate glucose by affecting the beta cells and alpha cells in the pancreas. Through DPP-4 inhibition, it works only when blood sugar is elevated to address diminished insulin due to beta-cell dysfunction and uncontrolled production of glucose by the liver due to alpha-cell and betacell dysfunction.

  • Apolipoprotein inhibitor

    Inherited Hypercholesterolemia is an inherited metabolic disorder resulting in an abnormal amount of cholesterol in the blood. It leaves patients at a high risk for adverse cardiovascular events, such as accelerated atherosclerosis and early heart attack. Dietary treatment seldom helps in these cases. A first-in-class medicine in development is an inhibitor of the protein, apolipoprotein, which plays a pivotal role in the production of low-density lipoprotein (LDL) or “bad” cholesterol. LDL is a type of protein that transports cholesterol and triglycerides from the liver to tissue in the body.



View additional resources to learn more about medicines currently in development to treat diseases that impact children: