The U.S. Food and Drug Administration granted rare pediatric disease designation to GBT440, a once-daily, oral, small-molecule, “anti-sickling” hemoglobin modifier, for the treatment of children with sickle cell disease (SCD).
GBT440 is being evaluated in the phase III Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization (HOPE) trial of patients with SCD ages 12 or older, as well as the HOPE-KIDS 1 study, an open-label, single- and multiple-dose trial for patients ages six to 17.
The FDA previously granted GBT440 orphan-drug and fast-track designations. GBT440 is also designated as an orphan medicinal product by the European Commission and is included in the European Medicines Agency’s Priority Medicines program.
Source: Global Blood Therapeutics press release, September 5, 2017.