Global Blood Therapeutics’ sickle cell disease drug succeeds in trial

(Reuters) – U.S. biotech company Global Blood Therapeutics Inc said on Wednesday its experimental drug met the main goal of a late-stage trial in patients with sickle cell disease, a potentially life-shortening inherited blood disorder.

A statistically significant number of patients on the treatment, voxelotor, showed a rise in levels of hemoglobin – a protein in red blood cells that carries oxygen throughout the body – compared to a placebo.

The data was from the first phase of the late-stage trial, and the company said it was in talks with the U.S. Food and Drug Administration on potential accelerated approval of the drug based on the main goal.

Sickle cell disease affects about 100,000 Americans and in 1 in 13 black or African-American babies are born with the trait, the Centers for Disease Control and Prevention estimates.

Mandatory screenings in the United States help diagnose newborns at birth and patients are currently treated with hydroxyurea, an old cancer drug, and antibiotics like penicillin to prevent infections.

The only potential cure at present is a bone marrow transplant but the procedure presents a risk of infection, infertility and death.

The company said it plans to present results from the study at a medical meeting later this year.

Reporting by Manas Mishra and Tamara Mathias in Bengaluru; Editing by Maju Samuel

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