Endari Approved for Sickle Cell Disease
July 7, 2017 – The U.S. FDA has approved Endari™ (L-glutamine) oral powder, manufactured by Emmaus Life Sciences, Inc., for treatment of sickle cell disease. Sickle cell disease creates deficiencies in the amino acid L-glutamine. Endari acts as a replacement for this amino acid, and can decrease complications of sickle cell disease, including acute attacks (sickle cell crises), for patients as young as five years old. In clinical trials that lasted 48 weeks, patients on Endari had fewer crises, emergency room visits, and hospitalizations when compared to patients on a placebo. They also reported having less pain.
Sickle cell disease is a genetic condition that deforms red blood cells into a characteristic crescent or sickle shape. The damaged cells become less flexible, and as they become trapped in blood vessels, blood flow is decreased, which limits the amount of oxygen reaching body tissues. Frequent complications of sickle cell disease include anemia, fatigue, jaundice, and swollen hands and feet. Crises can be triggered by dehydration, extreme heat or cold, illness, and stress. Crises may also occur spontaneously and unpredictably. Internal organs eventually sustain significant damage as a result of sickle cell disease. About 100,000 Americans have the disease, which is most common among individuals of African, southwest Asian, Middle Eastern, Hispanic, or southern European heritage.
Endari powder is taken mixed with eight ounces of a cold or lukewarm drink, or about four ounces to six ounces of soft food, such as pudding or applesauce, and then consumed right away. Recommended dosing is a total of 10g to 30g per day, based on body weight, and split across two doses. Emmaus Life Sciences has not yet announced launch or pricing plans for Endari.
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