FDA approves first medicine targeting root cause of sickle cell disease

FDA approves first medicine targeting root cause of sickle cell disease
Credit: Shutterstock.com/ Kateryna Kon

The US Food and Drug Administration has approved the first medicine specifically targeting the root cause of sickle cell disease (SCD) in adults and children 12 years and older.

Oxbryta (voxelotor), an oral therapy taken once daily, directly inhibits sickle haemoglobin polymerization, the root cause of SCD.

The medicine is expected to be available through GBT’s specialty pharmacy partner network within two weeks, developer Global Blood Therapeutics (GBT) said.

“Uniquely developed from inception to treat SCD, Oxbryta embodies GBT’s commitment to develop and deliver innovative medicines for patients with overlooked, life-limiting chronic diseases,” said Ted W. Love, President and CEO of GBT.

The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in haemoglobin levels, accompanied by reductions in red blood cell destruction (haemolysis).