Global Blood Therapeutics publishes sickle cell study data

Global Blood Therapeutics announced that data from its Phase 1/2 study of voxelotor in patients with sickle cell disease were published online in Blood, a peer-reviewed publication of the American Society of Hematology. Voxelotor is being developed as a potentially disease-modifying therapy for SCD. GBT intends to submit a New Drug Application, or NDA, for voxelotor under an accelerated approval pathway to the FDA later this year. “The findings from the Phase 1/2 study and the open-label extension played an important role in our development of voxelotor. We believe voxelotor has the potential to become a standard-of-care therapy in SCD, as evidenced by its ability to reduce anemia, hemolysis and sickling,” said GBT CEO Ted Love. “Additionally, I’m pleased that we have reached agreement with the FDA on both the accelerated approval pathway for voxelotor and for transcranial doppler (TCD) flow velocity as an acceptable primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction. We plan on initiating our TCD study later this year. I look forward to our upcoming pre-NDA meeting with the FDA for voxelotor, which will primarily focus on the format of our planned NDA filing in the second-half of this year.”
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