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The Orphan Drug Boom: Gold Rush Or Flash In The Pan?

Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.

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...

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