With all due respect Lilly, how do you get Billions of $ from Pitt-Hopkins when only 500 people worldwide have ever been diagnosed with it?
And only 1% of those with autism have Phelan-McDermid Syndrome.
Let's not get carried away.
It's clear the approach will be get approved for these and then push for off-label treatments imo.
Ann: FDA grants Orphan for both Phelan-McDermid and Pitt Hopkins, page-21
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