New treatment for sickle cell disease


The U.S. Food and Drug Administration approves Endari (L-glutamine oral powder) for patients with sickle cell disease to reduce severe complications associated with the blood disorder.

The approval was based on a randomised trial of Endari versus placebo in patients age five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial.

Relative to the patients treated with placebo, patients treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days). Also the patients who received Endari had fewer occurrences of acute chest syndrome compared with patients who received a placebo (8.6% vs. 23.1%).

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

Endari is marketed by Emmaus Medical Inc.

For more details on the approval click FDA Endari approval

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