Medicines

First sickle cell drug approved in two decades

Tuesday, 11 Jul 2017


The FDA approved the first new treatment for sickle cell disease in nearly 20 years. The new drug, Endari, (L-glutamine oral powder) was studied in a randomised placebo controlled 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 who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo, fewer hospitalisations for sickle cell pain , and fewer days in hospital.  Patients who received Endari also had fewer occurrences of acute chest syndrome compared with patients who received a placebo.

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