Nearly 450 Innovative Medicines in Development For Neurological Disorders

WASHINGTON, D.C. (July 30, 2013) – America’s biopharmaceutical research companies are developing 444 new medicines to prevent and treat neurological disorders, according to a new report released today by the Pharmaceutical Research and Manufacturers of America (PhRMA).

phrma report neurological disorders

There are more than 600 neurological disorders that strike millions of Americans each year. These disorders inflict great pain and suffering on millions of patients and their families, and cost the U.S. economy billions of dollars annually. They range from well-known disorders such as epilepsy, multiple sclerosis and Parkinson’s disease, to more obscure conditions such as Duchenne muscular dystrophy and Friedreich’s ataxia.

“Despite the incredible complexity of neurological disorders, biopharmaceutical scientists have delivered significant advances for patients in recent years, including new medicines for multiple sclerosis and Huntington’s disease,” said PhRMA President and CEO John J. Castellani. “The nearly 450 medicines in the pipeline have the potential to bring us ever closer to our shared goal of improving the health and extending the lives, of patients facing these debilitating disorders.”

These potential medicines – all in human clinical trials or under review by the Food and Drug Administration (FDA) – are diverse in scope. They include:

  • 82 medicines for Alzheimer’s disease, which afflicts more than 5 million Americans
  • 82 for pain – 100 million U.S. adults experience chronic pain
  • 62 for brain tumors – nearly 70,000 Americans are diagnosed each year with a primary brain tumor
  • 38 for multiple sclerosis, which afflicts an estimated 500,000 Americans
  • 25 for headache, a condition that affects about 28 million Americans
  • 28 for epilepsy and seizures, which impacts more than 3 million Americans
  • 27 for Parkinson’s disease, which affects as many as 1.5 million Americans

Of the 444 medicines in development, many represent novel, exciting scientific approaches  to target diseases, such as a gene therapy to restore neuronal function in Alzheimer’s patients, targeted therapies for neuromuscular disorders and gene therapy to restore cells damaged in Parkinson’s patients.

Current research has the potential to increase the quality of life and delay cognitive decline for patients diagnosed with neurological disorders. For example, scientists are beginning to understand more about the genes that affect the development of Alzheimer’s disease. Such discoveries could lead to new research pathways to help find a way to slow, delay or reverse the effects of the disease.  According to the Alzheimer’s Association, a treatment that delays the onset of Alzheimer’s by five years could reduce the cost of care for the disease by $447 billion a year in 2050.

Over the past decade, scientific advances and new technologies have dramatically changed how medicines are discovered. This new information is critical to the development of new treatments for neurological disorders.  Greater knowledge of how diseases work at the genetic and molecular level has allowed researchers to pursue new targets for therapy and better predict how certain biopharmaceuticals will affect specific subpopulations of patients.

“Collaboration among all partners in the medical innovation ecosystem – including patients – is critical to help advance scientific understanding of some of the most complicated neurological disorders,” added Castellani. “Even with all the advancements made to date – and the hope provided in the pipeline – we have much to learn about how the human brain functions. Together, we can make it happen.”

The Medicines in Development for Neurological Disorders report and other supplementary materials are available on PhRMA’s website at


The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested approximately $550 billion in the search for new treatments and cures, including an estimated $48.5 billion in 2012 alone.

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