New Sickle Cell Disease Treatment Gets FDA Approval

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This marks the first approval for a sickle cell disease treatment in nearly 2 decades.
This marks the first approval for a sickle cell disease treatment in nearly 2 decades.

The Food and Drug Administration (FDA) has approved Endari (L-glutamine oral powder; Emmaus Medical) to reduce acute complications of sickle cell disease in patients aged ≥5 years. This marks the first approval for a sickle cell disease treatment in nearly 2 decades. 

The safety and efficacy of Endari were evaluated in a randomized clinical trial involving patients aged 5–58 years with sickle cell disease who had ≥2 painful crises within 12 months prior to trial enrollment. Study patients received either Endari or placebo for 48 weeks. The results showed Endari-treated patients averaged less hospital visits for pain treated with a parenteral narcotic or ketorolac (sickle cell crises) vs patients given placebo (median 3 vs median 4). Patients in the Endari group also had fewer hospitalizations for sickle cell pain (median 2 vs median 3), and shorter length of hospital stay (median 6.5 days vs median 11 days). There were also a lower incidence of acute chest syndrome in Endari-treated patients vs placebo (8.6% vs 23.1%).  

Common adverse effects associated with Endari included constipation, nausea, headache, abdominal pain, cough, extremity pain, back pain, and chest pain.

Endari, an amino acid, was previously granted Orphan Drug and Fast Track designations from the FDA. L-glutamine may improve the NAD redox potential in sickle red blood cells through increasing the availability of reduced glutathione.

Endari will be available as packets containing 5 g of L-glutamine powder in 60-count cartons.

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