i thought orphan drug designation was more about the actual condition being rare and not so profitable and this designation just gave the company a chance to make more money in the long term..... provided it was the only treatment or better than existing
so it would apply to any drug treating Retts
thats my thinking, not sure if i am right or wrong
http://www.fda.gov/ForIndustry/Deve...pplyforOrphanProductDesignation/ucm240819.htm
FDA Grants Orphan Drug Designation, page-10
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