In the 25 years since FDA implemented the landmark Orphan Drug Act, providing incentives to drug makers to develop drugs for rare diseases, the area of drug development has largely been left to nimble biotechs and specialty pharmas.

But faced with growing generic competition in primary care markets, inadequate pipelines and increased regulatory and reimbursement challenges for new drugs, Big Pharma is showing renewed interest in the orphan drug market. Pfizer recently signed a deal with Protalix BioTherapeutics for rights to the enzyme replacement therapy taliglucerase for the ultra orphan indication Gaucher's disease, while GlaxoSmithKline partnered with Prosensa for a drug that would treat just a small subset of patients with Duchenne muscular dystrophy, already an orphan indication. Novartis recently launched Ilaris for a rare auto-inflammatory disorder that affects only 300 to 500 people in the U.S.

One incentive for Big Pharma is that the orphan drug business model has been shown to be a profitable one. Orphan drugs can command premium prices - in some cases up to hundreds of thousands of dollars a year - when those drugs offer an effective treatment for a rare and serious disease. And, while it's difficult to build blockbusters in the orphan drug space it's not impossible. Novartis' leukemia drug Gleevec is like a carrot dangling within the grasp of Big Pharma, an example of just how successful an orphan drug can be, when the unmet need it addresses is high and the therapy effective.

Gleevec first launched in 2001 in what was then considered a niche market, chronic myeloid leukemia. It has since grown into Novartis' second best-selling drug with sales close to $4 billion in 2009 (see chart: " ¹ Gleevec Rising Trend "). Part of the sales growth stems from expansion of the CML market, but it has also come from the 10 additional indications Gleevec is now approved for. Gleevec's rise to blockbuster status was fast too, coming just after its third full year on the market. Whether Eli Lilly will be able to say as much for Effient or Johnson & Johnson for Simponi, two drugs that launched in 2009 in traditional blockbuster categories, appears doubtful

But blockbuster orphans are anomalies, and ones that grow as swiftly as Gleevec even more so.

Genzyme's Gaucher's disease drug Cerezyme is another notable exception, but that drug took 13 years to cross the $1 billion threshold after its launch in 1994. It doesn't seem likely investors in the drug giants will be as patient waiting for a drug's sales to ramp up. Plus, selling drugs to niche patient populations requires a vastly different marketing tact then that used by Big Pharma, with its massive commercial organizations fueled by share of voice and boots on the ground. Who the winners will be when it comes to Big Pharma's latest diversification strategy remains to be seen, but it could be patients. (For more information on Big Pharma's move into the orphan drug market, see ² Also see "Orphaned No Longer: Big Pharma Embraces Drugs for Niche Markets" - In Vivo, 1 February, 2010. IN VIVO, February 2010).

- Jessica Merrill (³ j.merrill@elsevier.com)

Gleevec's Rapid Rise To Blockbuster Status

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