566 New Drugs in Pipeline for Orphan Diseases

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9 May 2016. A report by an industry group says 566 drugs designed to treat rare diseases are now in clinical testing by pharmaceutical companies. The report, prepared by Pharmaceutical Research and Manufacturers of America, or PhRMA, and the ALS Association, credits many of these new drugs to incentives in legislation encouraging research and development on treatments for rare diseases.

These 7,000 rare disorders, also known as orphan diseases, each affect less than 200,000 individuals in the U.S. But taken together, says the report, orphan diseases affect about 30 million, or 1 in 10, Americans. Because of the small numbers — sometimes just a few hundred people — affected by each disease, diagnostics and development of new drugs are difficult. Few clinicians have experience with these diseases, and the biological pathways for treating the disorders are often complicated. In addition, the small numbers of potential patients make it difficult to recruit participants for clinical trials.

The report indicates that despite these challenges, 566 new drugs are in clinical trials or under consideration by regulatory authorities, as of mid-April 2016. About a quarter (27%) of the drugs, 151 of the total, aim to treat rare forms of cancer, with more than half of that group designed for rare blood-related cancers. Another 148 new drugs, again about a quarter (26%) of the total, are treatments for genetic disorders, such as cystic fibrosis and spinal muscular atrophy. In addition drug companies have 31 therapies in the pipeline for rare bacterial infections and other infectious diseases, as well as 25 new treatments for rare autoimmune disorders.

Of the total, 38 new drugs are being tested for neurological disorders, including amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease; ALS Association collaborated with PhRMA on the report. ALS is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. The report lists 9 of those 38 therapies specifically for ALS in clinical trials, including treatments using stem cells.

The report points to the Orphan Drug Act of 1983 as a key factor in encouraging new drugs for rare diseases. The act gives the U.S. Food and Drug Administration the authority to designate an orphan drug, for treatments being developed targeting diseases affecting fewer than 200,000 Americans. Therapies, both drugs and biologics, designated as orphan drugs qualify for incentives such as tax credits for clinical trials and exemptions from marketing application fees.

In the past 10 years, says the report, FDA approved more than 230 new drugs for rare diseases, with nearly half (47%) of treatments approved in 2015 designated as orphan drugs. In the 1970s, the decade before the law was passed, fewer than 10 drugs for rare diseases were approved by FDA. Since passage of the Orphan Drug Act, FDA approved more than 500 orphan drugs.

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