- The FDA designates Global Blood Therapeutics' (NASDAQ:GBT -4.6%) lead product candidate GBT440 for Rare Pediatric Disease status for the treatment of sickle cell disease (SCD). The designation allows the company to receive a Priority Review voucher, if GBT440 is approved for the indication, that can be used for accelerated review of a future marketing application or it can be sold to a third party.
- GBT440 also has Orphan Drug and Fast Track status in the U.S.
- Previously: Enrollment underway in late-stage study assessing Global Blood's GBT440 in sickle cell disease (Jan. 20)