In DepthBiomedicine

Sickle cell drug raises hopes and doubts

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Science  20 Sep 2019:
Vol. 365, Issue 6459, pp. 1235
DOI: 10.1126/science.365.6459.1235

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Summary

Therapies for sickle cell disease, a grave genetic illness that afflicts about 100,000 Americans and millions of others globally, are woefully inadequate. So the news this month that the U.S. Food and Drug Administration (FDA) has launched a speedy review of a new drug that attacks the root cause of the disease has excited patients, along with some physicians who say they are cautiously optimistic. But other blood experts complain that the agency should not be rushing to approve a medicine that, in a pivotal clinical trial, failed to significantly reduce the episodes of intense pain that are the signature of the disease. Instead, FDA is considering approving the drug—voxelotor, made by Global Blood Therapeutics—based on significant improvements it caused in blood markers relevant to the disease. "Why we are approving a drug that hasn't shown that it has clinically meaningful benefit?" asks Robert Kruse, a pathology resident at Johns Hopkins Hospital in Baltimore, Maryland.

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