Can Voxelotor help SCD patients tide over blood shortage in COVID 19 season?

Sickle cell disease is a group of autosomal recessive diseases affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease.

Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.

Voxelotor (brand name Oxbryta) is developed by the Global Blood Therapeutics and recently the US FDA has also put the trial of this on fast track and designated the drug as breakthrough therapy.

In an interview with HCPLive®, Richard Drachtman, clinical section chief of Pediatric Hematology/Oncology at Rutgers Medicine, discussed how the recently-approved agent came into benefit for prescribing early into the pandemic. The mindset of a hematologist is to over-value donor blood. Once COVID-19 began to limit its availability further—especially in hard-hit areas —it became a responsibility to reduce the need for patient transfusions. "Use of voxelotor has allowed me to do that," Drachtman said.

"It gives you one more option of being able to avoid transfusions in this one population of patients," he said.

Voxelotor is an oral, once-daily therapy for patients with sickle cell disease (SCD). Voxelotor works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, voxelotor blocks polymerization and the resultant sickling and destruction of red blood cells.

The shortage of blood is acute but how far  Voxelotor helps SCD patients face this crisis, time only will tell.