Green Light for First Sickle Cell Rx in 20 Years Leave a comment


(RxWiki News) The US Food and Drug Administration (FDA) has approved the first medication to treat sickle cell disease in almost 20 years.

This new approval is called Endari (L-glutamine oral powder). The FDA approved it to treat patients 5 years old or older who have sickle cell disease. This medication is used to help patients reduce severe complications associated with the blood disorder.

For the estimated 100,000 people in the United States who have sickle cell disease, this new approval is another treatment option.

Sickle cell disease is a blood disorder that is passed on in families and is marked by red blood cells that are abnormally shaped. They are typically in a crescent, or “sickle,” shape, which restricts the flow in blood vessels and limits oxygen delivery to the tissues in the body. As a result, those with the disease may experience severe pain and organ damage.

This approval comes after a randomized trial showed that L-glutamine oral powder reduced the number of hospital visits and stays for pain and led to fewer days in the hospital when compared to a placebo. In addition, those who received L-glutamine oral powder had fewer episodes of a life-threatening complication of sickle cell disease called acute chest syndrome when compared to those who were given a placebo.

Common side effects include nausea, stomach pain, constipation, headache, cough and pain in the extremities, back and chest.

The FDA granted approval of Endari to Emmaus Medical Inc.


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