Targeted Therapies On FDA’s Orphan Designation Radar

As personalized medicine continues to grow, FDA is watching to see if there is a rise in orphan designations for products targeting a “smaller slice” of a common disease.

FDA is on the lookout for the effects of personalized medicine on the orphan drug program and the potential for all diseases to be defined in such a way that they become eligible for orphan status.

As the therapeutic principles behind personalized medicine continue to develop, there may be more drugs targeted at smaller, more specific...

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