BEACON TRANSCRIPT – The FDA approved a medicine which significantly reduces complications associated with sickle cell disease, a serious blood disorder. Endari is an oral powder which contains L-glutamine, and is destined for any patients older than five. This is a huge achievement, since it is the first time in 20 years when the FDA approves a treatment for the disease.
What is sickle cell disease?
Patients don’t develop sickle cell disease, since the condition is inherited. It affects their red blood cells, which are abnormally shaped like a sickle. This significantly reduces the amount of blood flowing through the vessels, so organs receive less oxygen than it is normal, also causing insufferable pain.
The National Institutes of Health discovered around 100,000 people living in the US suffer from this disease. It is most likely to occur among minority groups, and leads to a life expectancy placed somewhere between 40 and 60 years.
FDA supported the testing of the drug
Before Endari was approved, it was subjected to a randomized test with participants aged between 5 and 58. These participants had sickle cell disease and suffered at least two pain crises over the past 12 months. They were randomly assigned a treatment with either Endari and placebo and, for 48 weeks, were monitored to see how they react to the treatment.
Those patients who received Endari visited the hospital after pain crises less often than those with the placebo treatment, and also spent fewer days in the hospital. Also, they experienced acute chest syndrome less often, which is an extremely dangerous condition present in people who suffer from sickle cell disease.
Endari might come with certain side effects, but proved effective against the symptoms of sickle cell disease. Its trial and development was supported by FDA’s special program for drug designation for rare illnesses, which offers grants for testing and studies on its efficacy and safety.
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