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Global Blood Therapeutics Announces GBT440 Receives FDA Fast Track Designation for Sickle Cell Disease
“By granting Fast Track designation to GBT440, the
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
About Sickle Cell Disease (SCD)
Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS. In its deoxygenated state, HbS has a propensity to polymerize, or bind together forming long, rigid rods within a red blood cell (
Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act. We intend these forward-looking statements, including statements regarding the therapeutic potential of GBT440 and any other product candidates that we may identify and develop, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, those set forth in the prospectus for our initial public offering of common stock that was filed with the