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1 Rare Disease With an Array of Drugs

Keith Speights
October 2, 2012

French drugmaker Sanofi (NYSE: SNY  ) announced good news from its recent phase 3 trial of eliglustat tartrate, a drug targeting Gaucher disease. The trial met its primary endpoint of statistically significant improvement in spleen size at nine months for patients taking the drug. No safety concerns were raised during the study.

Crowded house?
Gaucher disease affects less than 10,000 people across the world. But prospective treatments for this rare disease aren't quite so rare these days.

Sanofi already maintains a significant presence in the market for Gaucher disease with its Cerezyme injection. Cerezyme is considered the standard of care for patients with type 1 of the disease.

However, Shire PLC (Nasdaq: SHPG  ) has made inroads with its Vpriv drug. Shire trumpeted clinical data in June that showed that patients receiving Vpriv experienced better lumbar spine bone mineral density than patients taking Cerezyme.┬áThe FDA granted orphan status to Vpriv in 2009, with the European Commission following in 2010. The drug i