HealthDay News — After a clinical trail, the US Food and Drug Administration (FDA) has approved Endari (L-glutamine oral powder) to treat patients with sickle cell disease.

Approval of Endari was based on a clinical trial that included patients aged 5 to 58 with sickle cell disease who had 2 or more painful crises within the 12 months before enrollment in the trial. 

During the 48-week study, patients who received the drug had fewer hospital visits for pain treated with a narcotic or ketorolac than those who received a placebo (median of 3 vs 4).


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They also had fewer hospitalizations for sickle cell pain (median of 2 vs 3), and fewer days in the hospital (median of 6.5 vs 11 days). Patients who received Endari also had a lower risk of acute chest syndrome than those who received a placebo (8.6 vs 23.1%).


Common side effects of Endari included constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain, and chest pain, according to the FDA.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products at the FDA’s Center for Drug Evaluation and Research, said in a statement. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

Reference

FDA Approved New Treatmant for Sickle Cell Disease [press release]. Silver Spring, MD: US Food and Drug Administration; July 7, 2017.

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