FDA Grants Breakthrough Therapy Designation to Global Blood Therapeutics’ Voxelotor for the treatment of sickle cell anemia

Voxelotor, a potential treatment for sickle cell anemia (SCA), has been given breakthrough therapy designation (BTD), Global Blood Therapeutics (GBT) recently announced.

Voxelotor (previously called GBT440) is a potential once-daily oral medicine for SCA patients. It was designed to increase hemoglobin’s binding of oxygen, which keeps red blood cells in their normal round shape, and helps prevent cells’ clumping and sickling.

GBT believes that the ability to repair hemoglobin function and improve oxygen delivery makes voxelotor a potential disease-modifying therapy in SCA. The medicine previously was granted fast track, orphan drug (given to promising products for the diagnosis and/or treatment of rare diseases) and rare pediatric disease designations.

The U.S. Food and Drug Administration (FDA) grants BTD to therapies for serious or life-threatening diseases if they show substantial improvement over existing medications in preliminary clinical results. The BTD label accelerates development and review of the new treatment.

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