Global Blood Therapeutics Announces GBT440 Receives FDA Fast Track Designation for Sickle Cell Disease

Global Blood Therapeutics (GBT)

, a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GBT440 for the treatment of sickle cell disease (SCD). "By granting Fast Track designation to GBT440, the FDA is acknowledging that SCD is a serious health problem that needs new treatment options. This gives us the opportunity to work closely with the FDA to accelerate development of GBT440 for this important unmet medical need," said Ted W. Love, M.D., chief executive officer of GBT. "We believe the unique ability of GBT440 to target the underlying mechanism of red blood cell sickling underscores its potential to modify the disease, rather than only treat its symptoms." GBT440 is an oral, once daily therapy for the treatment of SCD that works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT440 blocks polymerization and the resultant sickling of red blood cells (RBCs). With the potential to restore normal hemoglobin function, GBT440 may be capable of modifying the progression of SCD. Currently, GBT440 is being investigated in an ongoing Phase 1/2 clinical trial in both healthy subjects and SCD patients. The FDA's Fast Track process is designed to expedite the clinical development and submission of a New Drug Application (NDA) for medicines with the potential to treat serious or life-threatening conditions and address an unmet medical need (i.e., a condition whose treatment or diagnosis is not addressed adequately by available therapy). A drug program with Fast Track designation enables the company to have early and frequent communication with the FDA in the development and review of the product candidate, often leading to faster drug approval and access by patients.