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Global Blood's sickle cell therapy shows efficacy, tolerability in phase 2 trial

Global Blood Therapeutics Inc. said that its medicine voxelotor showed efficacy and tolerability in children with sickle cell disease, an inherited red blood cell disorder.

The South San Francisco, Calif.-based biopharmaceutical company is studying the drug in children ages 6 to 17 in an ongoing phase 2 trial dubbed Hope-Kids 1.

The study is divided into two portions: in one, patients received 600 milligrams of the drug, while in the other portion, subjects received 900 milligrams and 1,500 milligrams of the medicine.

Of the 22 patients who received 900 milligrams of voxelotor, 43% showed a hemoglobin response at a 24-week followup.

The sickle cell disease causes abnormality in the oxygen-carrying protein hemoglobin found in red blood cells.

Furthermore, 13 of 21 patients had reduced daily symptoms.

Voxelotor was well-tolerated by the patients with no drug-related discontinuations.

In addition, the company said that the effect of voxelotor in children receiving a single 600-milligram dose was higher than in adolescents or adults, suggesting that lower doses should be evaluated for future clinical trials in that patient population.

In January, voxelotor received the U.S. Food and Drug Administration's breakthrough therapy designation.