The top 10 or­phan drugs in the late-stage pipeline

Or­phan drugs have come in­to the spot­light, scar­ing up head­lines in­volv­ing big prices and small pa­tient pop­u­la­tions. Some com­pa­nies have been ac­cused of prof­i­teer­ing by ma­nip­u­lat­ing the FDA’s rules on this — hel­lo, Marathon — but or­phan ther­a­pies re­main a very close­ly watched are­na with some ma­jor new prospects com­ing through the late-stage pipeline. And some of these drugs are of­fer­ing pa­tients re­al hope for the first time.

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