Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for GBT440 for the treatment of patients with sickle cell disease (SCD). GBT440 is being developed as a potentially disease-modifying therapy for 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.”
The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics that are being developed to address rare diseases or disorders that affect fewer than 200,000 people in the U.S. With orphan designation, GBT qualifies for various incentives, including tax credits for qualified clinical trials and market exclusivity upon regulatory approval.
