Critical needs for patients – and business – met with orphan drug research

Industry experts estimate that orphan drugs bring in about $40 billion in annual sales. With drug companies facing devastating patent expirations and new drug opportunities drying up in existing markets, orphan drug development has become an enticing solution.

FDA and Congress have further lured pharma companies to this niche sector with various incentives. The Orphan Drug Act of 1983 provides seven-year market exclusivity, tax breaks, and regulatory help for companies developing new drugs for orphan diseases. Since its introduction, more than 2,100 compounds have been submitted for orphan status in the United States and more than 350 have received designation. FDA is urging large drug makers, such as Roche and Johnson & Johnson, to determine if existing medicines may be able to help neglected disorders. Drugs with orphan drug designation are likely to receive fast-track status.

Despite the small patient pools they are being developed for, the most successful orphan drugs have more than recouped R&D investment for their developers. Merck's brain cancer drug Temodar was originally approved in 1998 for treating the orphan indication anaplastic astrocytoma, has since been approved for other brain cancer forms, and generated sales of more than $1 billion in 2009. Genzyme Corp., the leader in the orphan drug sector, realized $793 million in sales in 2009 with its Gaucher disease drug Cerezyme. Genzyme's success with Cerezyme and other rare disease treatments is likely the driver behind sanofi-aventis' attempted takeover of the company. More large companies are entering the orphan drug sector. Earlier this year, both GlaxoSmithKline and Pfizer Inc. opened research units dedicated to developing new biologics to treat rare diseases.

The most transformational devices to be developed in recent years are drug-eluting stents. These devices improved procedural quantities in two major cardiology markets. Another device success, Vitrasert, is an intraocular sustained delivery system for AIDS-related cytomegalovirus retinitis. This device has been very effective for this indication and, more importantly, has lead to commonplace use of intraocular drug delivery for other indications, including Lucentis for age-related macular degeneration.
     
A newly released PharmaLive report listed many developments in the orphan drug market, including partnerships, acquisitions, and investments that could be of interest to you.