Rare Disease Report

FDA Grants Orphan Drug Designation to Sickle Cell Treatment

JANUARY 05, 2016
Rebekah Harrison
The FDA has granted orphan drug designations to Global Blood Therapeutics’ GBT440 for the treatment of patients with sickle cell disease (SCD).
Sickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, mostly African Americans.  A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.
GBT440 prevents sickling by blocking sickle hemoglobin (HbS) polymerization by maintaining HbS in the oxygenated state. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may be capable of modifying the progression of SCD.
“Receiving orphan drug designation, along with the previously announced Fast Track designation, are important milestones in our regulatory strategy for GBT440 and highlight the FDA’s agreement that the SCD community faces a critical need for new treatments,” said Ted W. Love, M.D., chief executive officer of GBT. “We continue to believe that GBT440 has the potential to become the first mechanism-based and disease-modifying therapeutic for this grievous disease and look forward to sharing full results from our Phase 1/2 trial and potentially initiating a pivotal trial in adult patients with SCD in 2016.” 
Currently, GBT440 is being investigated in an ongoing Phase 1/2 clinical trial in both healthy subjects and SCD patients.


Global Blood Therapeutics Receives FDA Orphan Drug Designation for GBT440 in Sickle Cell Disease [news release]. San Francisco, CA; Global Blood Therapeutics: January 4, 2015.

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