IN The News….

Voxelotor (Oxbryta) attacks the root cause of Sickle Cell Disease

The US Food and Drug Administration (FDA) has approved VOXELOTOR, in oral tablets (Oxbryta) in the treatment of sickle cell disease (SCD) in adults and children older than 12. This is a novel therapy that could change the course of this disease.

“Voxelotor” attacks the root cause of SCD in inhibiting directly the hemoglobin polymerization. With this drug, sickle cells are less likely to bind together and deform the red cells to convert in sickled shape unable to carry enough Oxygen to the tissue. They do have a shorter life span creating a low hemoglobin level.

This therapy brings a new form of treatment for patients with serious and life-threatening condition. A clinical trial was conducted on 274 patients with sickle cell disease with “Voxelotor” and a placebo which lead to significant improvements in hemoglobin levels while the red blood cells were protected from hemolysis. It took ten years to offer this treatment option.

It takes 6 months (24 weeks) to realize that 51.1% of patients receiving the medication were able to improve their hemoglobin level to 1g/dL. The medication comes with side effects like headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).

The results were reported and published in the New England Journal of Medicine and the Med-scape Medical News in June 2019. Elliot Vichinsky MD, director of hematology/oncology at USCF Benioff Children Hospital in Okland, CA was caught saying that “Every person with SCD experiences hemoglobin polymerization and suffers from varying severity of anemia and hemolysis, then should be a candidate to receive such medication.

“Vexoletor” is a new medication that significantly improves hemoglobin levels and reduces the anemia and hemolysis that is seen in this genetic condition. It can be part of your armamentarium in the treatment of sickle cell disease. Also earlier in the month, Adakveo was also approved. This drug reduces as well the frequency of vaso-occlusive crisis in patients older than 16, with sickle cell disease. The news was reported by Medscape Medical News. 

Maxime Coles MD


1-    Article from Megan Brooks, November 2019

2-    Vichinsky, E Hematology Department, Children Hospital in Okland CA