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Global Blood Therapeutics' blood disorder drug accepted for US FDA review

Global Blood Therapeutics Inc.'s experimental treatment of sickle disease voxelotor has been accepted for review by the U.S. Food and Drug Administration.

Sickle cell disease is a lifelong inherited blood disorder caused by a mutation in the oxygen-carrying blood component called hemoglobin. As a result, patients experience a type of anemia that can lead to organ damage, severe pain and premature death.

Voxelotor, which would be an oral tablet, will be reviewed by the FDA under the priority protocol that reduces the assessment duration to six months. The South San Francisco, Calif.-based biotechnology company will receive a decision from the U.S. regulator by Feb. 26, 2020.

The drug is specifically meant to reduce the risk of stroke in patients by increasing hemoglobin's affinity for oxygen as opposed to giving them blood transfusions.

Voxelotor's application is based on data from a phase 3 study called Hope in which the drug raised the level of hemoglobin in 58% of the patients after 12 weeks of treatment compared to 9% treated with placebo. The trial is ongoing in sickle cell disease patients aged 12 years and older.

The FDA granted voxelotor breakthrough-therapy designation in January 2018 and agreed to provide an accelerated approval pathway for the blood disorder drug in December the same year.

Global Blood Therapeutics' share price was up 12% following the news to $53.21 as of 11:28 a.m. ET on Sept. 5, 2019.