Most of the orphan drugs on the market have been approved for a single indication, but close to 50 compounds have received regulatory clearance in multiple rare diseases. With seven separate orphan drug approvals, imatinib (Gleevec; Novartis) is one of the most commercially successful drugs for treating rare diseases. Sales of imatinib reached US$4.65 billion in 2011. In 2006–2008, 16 orphan therapeutics made the top 200 list for US drug sales, with annual sales ranging from $200 million to over $2 billion.

It is now widely recognized that rare diseases provide attractive niche opportunities for biopharmaceutical companies. Orphan drugs offer advantages such as faster development timelines, lower research and development expenses, a higher likelihood of clinical and regulatory success, premium pricing, lower marketing costs and a lower risk of generic competition. According to the Tufts Center for the Study of Drug Development, companies reported that 22% of their programmes designated as orphan drugs led to approvals between 2000 and 2009, whereas clinical approval success rate for mainstream drugs was 16%. Overall, in the past few years, medicines for rare conditions accounted for over 35% of the new drugs approved by the US Food and Drug Administration (FDA).

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