FDA approves sickle cell treatment, the first in almost two decades

FDA approves sickle cell treatment, the first in almost two decades

FDA approves sickle cell treatment, the first in almost two decades

The FDA approved a medicine which significantly reduces complications associated with sickle cell disease, a serious blood disorder.

"The approval of Endari is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for almost 20 years and which now, for the first time ever, has a treatment option for children", said Yutaka Niihara, MD, MPH, chairman and chief executive officer of Emmaus Life Sciences.

What is sickle cell disease? It affects their red blood cells, which are abnormally shaped like a sickle. "All are impacted if one has to be hospitalized frequently for something like pain". According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease.

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Persons with sickle cell disease inherit two abnormal hemoglobin genes, one from each parent. This is a huge achievement, since it is the first time in 20 years when the FDA approves a treatment for the disease. It is most likely to occur among minority groups, and leads to a life expectancy placed somewhere between 40 and 60 years.

Endari might come with certain side effects, but proved effective against the symptoms of sickle cell disease. "We are able to provide vaccinations, penicillin, close medical management".

That's what makes Endari exciting, she says. Endari reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme that has been identified as the primary regulator of oxidation. Endari works by increasing the levels of L-glutamine in the blood.

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The FDA says Endari was studied in a random trial of patients from ages five to 58 years old with sickle cell disease who had more painful crises within a year before the trial.

Endari had received an orphan drug designation for this use.

"We clearly have much room for improvement in what we can provide for people with sickle cell disease and while we are clearly very excited to see another drug reach the marketplace, we need more", Thompson said.

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