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L-glutamine Oral Powder Significantly Reduces Acute Complications of Sickle Cell Disease


Approved by the US Food and Drug Administration (FDA) in July 2017 to reduce the acute vaso-occlusive pain complications of sickle cell disease, L-glutamine oral powder (Endari) was the first drug approved for the rare blood disease in the 20 years since the approval of hydroxyurea.

Now, the New England Journal of Medicine (NEJM) has published the 48-week phase 3 clinical trial results that supported the FDA approval. From the data acquired in the phase 3 trial, investigators concluded that the median number of pain crises in children and adults with sickle cell disease was lower among those who received oral therapy with l-glutamine, administered alone, or with hydroxyurea, compared with those who received placebo, with or without hydroxyurea.

“The idea that this [publication] comes a year after [approval] really reflects the vigor of the review process and shows NEJM really did its job of assuring the public interest that L-glutamine, along with the findings of the study, are real and significant,” Darrell W. Harrington, MD, MACP, chief medical officer at Emmaus Life Sciences, told Rare Disease Report®.


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