It's not easy to develop treatments for rare diseases, but it is hard to argue with the rewards. Prior to its acquisition by Sanofi (NYSE: SNY ) , Genzyme had already established itself as an important player in enzyme replacement therapies, and Shire (NASDAQ: SHPG ) and Alexion (NASDAQ: ALXN ) have both gone on to find meaningful success with treatments that serve tiny patient populations, but carry huge price tags.
BioMarin (NASDAQ: BMRN )
very much deserves to be in this conversation, as the company has one
of the broadest portfolios and pipelines for rare diseases. BioMarin's
pipeline definitely has clinical risk and I don't think investors should
just assume that payers will always go along with the pricing these
companies want. The biggest issue may be overall expectations, though,
as BioMarin is going to have to become extremely profitable and deliver
exceptional revenue growth just to meet current expectations.
Read the full article here:
BioMarin Pharmaceutical, Inc.: Is This Orphan Drug Specialist Overvalued?
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