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Biotech / Medical : Blueprint Medicines

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To: tuck who wrote (11)12/3/2018 8:25:01 AM
From: atticus4paws   of 12
 
Thanks Tuck! A couple of things I'm wondering...

Will they design the P2 for possible approval? It should be possible to demonstrate efficacy with smallish numbers, but as it will be a chronic drug, does long term safety become the dominant consideration?

Regarding prevalence (my mind gets turned around with incidence sometimes - not today)...

If we assume that BPMC has estimated correctly in the U.S. (~8500 across all SM types) and that number will be fairly steady with population changes and they capture the entire market, wouldn't it still necessitate a hefty price tag to make this drug much of an earner in SM?
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