If you mentioned “orphan drugs” to a pharma audience even three years ago, just a handful of drug manufacturers would likely have come to mind: the pioneer Genzyme Corp., naturally, along with comrades-in-arms Shire PLC, BioMarin Pharmaceutical Inc., and Actelion Pharmaceuticals Ltd. Well, things have changed.
In the last 18 months alone, Genzyme was acquired by Sanofi[See Deal], Pfizer Inc. launched Elelyso (taliglucerase) for the rare condition Gaucher’s disease, and GlaxoSmithKline PLC initiated a Phase III trial for what could potentially be the first drug to treat a small subset of patients with Duchenne muscular dystrophy. And it’s not just Big Pharmas that are blazing a trail into rare diseases, an area of drug development that refers to diseases that afflict fewer than 200,000 patients in the US, but are often far smaller. Alexion Pharmaceuticals Inc
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