Voxelotor in Sickle Cell Disease

July 29, 2020

How has the new agent fared in its indicated patients in its first year on the market?

In late November last year, the US Food and Drug Administration (FDA) granted approval to voxelotor (Oxbryta), a deoxygenated sickle hemoglovin polymerization inhibitor, for the treatment of sickle cell disease in patients aged 12 years and older.

The therapy was touted for its ability to reduce sickle cell binding, showing a 51.1% hemoglobin response rate among patients administered 1500 mg dose in the pivotal trial included in the FDA submission.

So, nearing 1 year as an available therapy in an at-need field, where does voxelotor stand now?

In an interview with HCPLive®, Richard Drachtman, MD, professor of Pediatrics and Clinical Section Chief of Pediatric Hematology/Oncology at Rutgers Medical, discussed his own excitement surrounding the original voxelotor approval—and his own experiences in prescribing and caring for patients with sickle cell.

“It’s been a very interesting time,” Drachtman told HCPLive. “We’ve been prescribing it since late December-early January, and we’re seeing some very good results.”

Drachtman explained he had his own “short list” of patients he anticipated would benefit most from the new therapy once it was marketed: those with the lowest hemoglobin counts and hemolytic characteristics.

Included among those patients were the 10% to 20% of all sickle cell disease patients with primary hemolysis—who are often subjected to burdensome blood transfusions.

Drachtman also set parameters for discussing voxelotor with potentially eligible patients based on hemoglobin levels. It’s now that he’s beginning to see the impact of the new therapy which he prescribed to patients 2-3 months ago.

“We’re seeing in the patients who had very low hemoglobin levels and were very hemolytic to start significant rises in hemoglobin and significant drops in bilirubin,” he explained. “We’re seeing them not needing transfusions anymore, or to the same degree. We’re seeing significant changes in the use of healthcare, as a result.”


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