For the first time in nearly two decades, the U.S. Food and Drug Administration has approved a new treatment option for sickle cell. Last month, the federal agency gave the green light to Endari (L-glutamine oral powder) for patients ages five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.

The safety and efficacy of Endari were studied in a randomized trial of patients ages 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. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac on average, compared to patients who received a placebo (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). Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

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