Global Blood’s drug might satisfy FDA, but some doctors are wary

A sickle-cell disease drug from Global Blood Therapeutics looks to be on the path to FDA approval, judging by new data released today, but whether the pill’s effects will actually improve patients’ lives remains the subject of debate.

As STAT’s Adam Feuerstein reports, the drug, called voxelotor, significantly increased patients’ hemoglobin levels in a late-stage trial. That, according to Global Blood, should be enough to qualify voxelotor for accelerated FDA approval.

But outside experts cautioned that boosting hemoglobin might not actually address the worst symptom of sickle-cell disease: episodes of severe pain that often land patients in the hospital. Global Blood doesn’t have data on whether voxelotor can do that.

“There is pressure on everybody to pay attention to sickle-cell disease and get more therapeutic options out there, but I don’t think this is a good reason to push through voxelotor’s approval if it doesn’t meet the bar of showing benefit for patients,”  said Dr. Sujit Sheth of Weill Cornell Medicine.

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