First Drug To Tackle SCD Cause, Not Symptoms, Emerges

Normal and sickled cells in circulation (USFDA)

‘The tide is turning against sickle cell disease. With two new weapons approved for #sicklecellwarriors in a week and 3 in 2 years, after a 20-year wait, a Golden Age of #SickleCell treatment has officially arrived!’

– Alexander Glaros, Paediatric Haematology-Oncology Fellow, Children’s Hospital of Michigan

The U.S. Food and Drug Administration has approved a new drug for SCD, one of a long line of drug therapies for the inherited blood disorder affecting an estimated 20 million people worldwide. To be sold under the brand name Oxbryta, the drug is from pharmaceutical giant, Global Blood Therapeutics Inc. and is for the treatment of SCD in adults and children aged 12 years or older.

Oxbryta is the second drug in days to win approval for sickle cell anemia, and the very first to target the underlying cause of the disease rather than symptoms. It’s approval comes just over one week after Novartis’ sickle cell drug, Adakveo, won regulatory clearance.

Known chemically as voxelotor, it works by preventing red blood cells from sickling, a deformation that restricts flow of oxygen in blood vessels, leading to severe pain and organ damage.

“Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” the FDA said in a statement.

Sickle cell disease remains most prevalent in sub-Saharan African countries, where many of the estimated 300,000 children born annually with the condition may die before the age of 5. The disease is also common in India.

The approval of vexolotor is a milestone in SCD care and management. Pediatric Hematology-Oncology Fellow at Children’s Hospital of Michigan, USA, Alexander Glaros, MD, was ecstatic about recent events in sickle cell drug research, stating –

‘The tide is turning against sickle cell disease. With two new weapons approved for #sicklecellwarriors in a week and 3 in 2 years, after a 20-year wait, a Golden Age of #SickleCell treatment has officially arrived!’

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