Rare Disease Report

FDA Approves Glutamine Powder for Sickle Cell Disease

JULY 06, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has approved Emmaus Life Sciences' Endari (L-glutamine oral powder) for the treatment of sickle cell disease.
More specifically, the powder formulation is approved for patients ages 5 years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
Sickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, most of whom are African Americans. A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke. Painful crises are a common feature of this condition and L-glutamine has been shown to reduce the number of crises in patients.
Per the FDA: "Endari is the first treatment approved for patients with sickle cell disease in almost 20 years," said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. "Until now, only one other drug was approved for patients living with this serious, debilitating condition."

The approval of Endari was largely based on a randomized trial of patients with sickle cell disease who had 2 or more painful crises within the 12 months prior to enrollment. Patients were given Endari (L-glutamine) or placebo and followed for 48 weeks. The study observed that patients receiving Endari had fewer sickle cell crises requiring hospitalization and pain treatment compared to placebo (3 vs 4), fewer hospitalizations for sickle cell pain (2 vs. 3), and fewer days in the hospital (6.5 days vs. 11 days).  Patients who received Endari also had fewer occurrences of acute chest syndrome compared to placebo (8.6% vs. 23.1%).
Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.
At the American Society of Hematology (ASH) annual meeting, Rare Disease Report spoke with Yutaka Niihara, MD, MPH, president of Emmaus Life Sciences, about sickle cell disease and the use of L-glutamine to treat patients.

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