New Sickle Cell Disease Treatment Gets FDA Approval
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%).
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.