FDA approves first drug to target the root cause of sickle cell disease

By Vassia Barba

- Last updated on GMT

(Image: Getty/Dr_Microbe)
(Image: Getty/Dr_Microbe)

Related tags sickle cell FDA approval Fda Global Blood Therapeutics

Global Blood Therapeutics receives approval for Oxbryta, making it the first approved drug product that targets the root cause of sickle cell disease.

California, US-based biopharmaceutical company, Global Blood Therapeutics (GBT), announced it received approval by the US Food and Drug Administration (FDA) for Oxbryta (voxelotor), for the treatment of sickle cell disease in adults and children aged 12 years and older.

According to GBT, Oxbryta is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of sickle cell disease (SCD).

The one-daily, orally-administered treatment, is expected to be available through GBT’s specialty pharmacy partner network, starting December 2019.

The FDA granted accelerated approval to GBT for the drug product based on data from a Phase III clinical study, during which 51.1% of 274 patients with SCD presented significant improvements in hemoglobin levels and reductions in red blood cell destruction after 24 weeks of treatment.

SCD is a blood disorder that can cause life-threatening events, such as stroke and irreversible organ damage. According to the US Center for Disease Control and Prevention​, SCD affects an estimated 100,000 people in the US, as well as millions of people throughout the world.

Beverley Francis-Gibson, CEO of the American Sickle Cell Disease Association, commented that “after decades of waiting, we now have a treatment option that could change the course of this disease.”

Prior to its approval, Oxbryta was granted several acceleration designations by the FDA, including priority review, breakthrough therapy, fast track, orphan drug and rare pediatric disease designations.

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