GBT Announces Publication of Data from Phase 1/2 Study of Voxelotor in Patients with Sickle Cell Disease

Global Blood Therapeutics (NASDAQ:GBT) today announced that data from its Phase 1/2 study of voxelotor in patients with sickle cell disease (SCD) 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 (NDA) for voxelotor under an accelerated approval pathway to the U.S. Food and Drug Administration (FDA) later this year.

As quoted in the press release:

“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 Ted W. Love, M.D., president and chief executive officer of GBT. “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.”

As reported in the Blood article, “A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease,” the safety, tolerability, pharmacokinetic and pharmacodynamic properties of voxelotor were demonstrated in patients with SCD. Thirty-eight patients with SCD received voxelotor at doses of 500, 700 or 1000 mg per day or placebo for 28 days; 16 patients received voxelotor at doses of 700 or 900 mg per day or placebo for 90 days.

Click here to read the full press release.