BioMarin targets cancer as an orphan

BioMarin Pharmaceutical Inc. wants to do for cancer what it’s done for rare diseases.
After transforming the drug-development world with its focus on diseases with only a few thousand patients, BioMarin is using that same model to tackle cancer in subsets of patients. So instead of working on a drug aimed at the full population of ovarian cancer patients — many of whom won’t benefit from the treatment — it is zeroing in on a much smaller group with a specific genetic mutation.
“It turns cancer into an orphan disorder,” said BioMarin CEO Jean-Jacques Bienaime.
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