A new sickle cell drug could soon get U.S. approval. But does it work?

Science | AAAS | 9/17/2019 | Staff
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A new sickle cell drug could soon get U.S. approval. But does it work?

A new drug for sickle cell disease, a grave genetic malady that afflicts an estimated 100,000 people in the United States alone and has no truly effective therapy, should be cause for rejoicing. Instead, what could be a rapid march to approval for voxelotor, which acts directly on the mutant protein that causes the disease, has sparked a dispute over the U.S. Food and Drug Administration’s (FDA’s) efforts to evaluate an urgently needed therapy.

Month - Drug - Developer - Global - Blood

This month, the drug’s developer, Global Blood Therapeutics (GBT) in South San Francisco, California, revealed that FDA has launched a “priority review” of voxelotor and does not plan to have a group of external experts provide advice on the matter before a 26 February 2020 deadline. Critics of the drug expect FDA will give a green light to the therapy, and they say approval would be premature. The drug improves “surrogate” endpoints that can be measured in lab tests but has not yet been proved to reduce the disease’s symptoms. “Why are we approving a drug that hasn’t shown that it has clinically meaningful benefit?” asks Robert Kruse, a pathologist at Johns Hopkins Hospital in Baltimore, Maryland, who treats sickle cell patients.

Sickle cell disease originated in Africa, where it’s estimated to prematurely kill 50% to 90% of the millions of people born with it. A mutation that alters the oxygen-carrying protein hemoglobin inside red blood cells is the culprit. Normal red blood cells are flexible enough to squeeze through small blood vessels. But the mutation causes hemoglobin to aggregate into rock-hard rods that give cells a sickle shape. Sickled cells clump, blocking blood vessels and triggering episodes of intense pain called vaso-occlusive crises. The cells are also brittle and prone to shattering, causing anemia...
(Excerpt) Read more at: Science | AAAS
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