Orphan drugs to see growth through 2018, report finds

NEW YORK — Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.

According to analysis by GBI Research, the orphan disease therapeutics market will see an increase in value through the decade, increasing from 2010's $2.3 billion to $6 billion in 2018. Currently, there are more than 6,000 orphan diseases recognized in the United States, defined as diseases affecting 200,000 or fewer Americans and including such conditions as Gaucher disease and Fabry disease. European health authorities recognize between 5,000 and 8,000 of them. The report focused on the United States, Japan, the United Kingdom, Germany, France, Italy and Spain.

The report found that the need for an improved and expanded selection of drugs would boost research and development, some of which already has been taking place: On Wednesday, the Food and Drug Administration approved Elelyso (taliglucerase alfa), a treatment made by Pfizer and Protalix BioTherapeutics for Type 1 Gaucher disease. Genzyme, now a subsidiary of French drug maker Sanofi, historically has been a leader in developing drugs for orphan diseases, but some of its drugs have experienced shortages due to manufacturing problems in recent years.

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