Voxelotor, marketed under the brand name Oxbryta™ (Global Blood Therapeutics Inc., GBT), has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients aged 12 years and older with sickle cell disease (SCD). Oxbryta™ is a once-daily pill that works by directly inhibiting sickle haemoglobin (Hb) polymerization, the main cause of SCD. The drug’s approval came three months earlier than expected and is based on results from the randomized, global Phase 3 HOPE trial (NCT03036813). Trial findings were published in The New England Journal of Medicine, showing clinically meaningful and statistically significant improvements in Hb levels, accompanied by reductions in red blood cell destruction (hemolysis). Briefly, 51.1% of patients receiving Oxbryta™ for 24 weeks at 1500 mg daily, its now approved dose, achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (n=274, P<0.001). Common side effects for patients taking Oxbryta™ were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever). In spite FDA Fast Tract designation, further clinical trials are required to verify and describe the drug's clinical benefit. Oxbryta™ also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. For more information, read GBT press release here and FDA press release here.