WASHINGTON -- The FDA said Friday it has : L-glutamine oral powder (Endari), indicated for reducing severe complications associated with the condition.
"Endari is the first treatment approved for patients with sickle cell disease in almost 20 years," Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in a statement.
The drug, produced by Emmaus Medical, was shown in placebo-controlled clinical trials to reduce both rates of hospital visits and admissions for acute sickle cell crisis as well as the incidence of acute chest syndrome.
An FDA advisory committee had recommended approval for Emmaus's L-glutamine in May.
L-glutamine is a precursor for nicotinamide adenine dinucleotide, which is depleted in sickle cell disease leading to impaired endogenous antioxidant activity. According to Emmaus's presentation at the May advisory committee meeting, "L-glutamine-induced increases in redox potential may reduce oxidative stress in [sickled] cells, resulting in decreased RBC adhesion, decreased vaso-occlusion, and thus, fewer painful sickle cell crises."