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Test Tubes

Voxelotor: Promising New Drug to Increase Oxygen Delivery and Reduce Sickle Cell Complication

On Thursday, September 5th, 2019 Global Blood Therapeutics (GBT)  announced receiving the U.S. Food and Drug Administration (FDA) new drug and priority review approval for Voxelotor. Formally named GBT440, Voxelotor "is being developed as an oral, once-daily therapy for patients with SCD" (sickle cell disease). The mechanism of action for this new treatment option is to improve hemoglobin-oxygen affinity and reduce polymerization of red blood cells (RBC). In doing so, Voxelotor is believed to reduce sickling of RBC's and increase oxygen delivery to cells and vital organs; thereby reducing the many complications associated with having SCD.

Although new on the scene to some, Voxelotor/GBT440, has been around since 2012 when GBT first began testing. In January of 2015, GBT began Phase I/II clinical trials in a small cohort of 8 individuals to test the "safety, tolerability, pharmacokinetics and pharmacodynamics" of the drug. This phase of the study would go on to enroll approximately 120 more subjects. By the end of 2015, the FDA granted Voxelotor Fast Track and Orphan Drug designation and by September 2016 GBT would announce its agreement with the FDA to begin the HOPE Study, a phase III clinical trial to test the efficacy and safety of the drug. In less than 24 months, GBT would report on data from Part A of the HOPE study with patients being treated with Voxelotor having "statistically significant increase in hemoglobin compared to baseline" and less vaso-occlusive crisis than participants on placebo.  

For the sickle cell community this represents significant progress in sickle cell research and treatment options. Not only is the sickle cell community gaining the attention of the federal government and pharmaceutical companies like GBT, they will also soon have the ability to choose from a list containing more then two drugs - Hydroxyurea (HU) which was approved in 1998 and Endari which was approved in 2017.  

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