The FDA has approved the first new treatment for patients with sickle cell disease in almost 20 years.

Endari (L-glutamine oral powder, Emmaus Medical Inc.) is indicated for patients 5 years of age and older with sickle cell disease to reduce severe complications associated with the blood disorder. "Until now, only one other drug was approved for patients living with this serious, debilitating condition,” said Richard Pazdur, MD, 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.

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent, or "sickle," shape). This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African-Americans, Latinos, and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is about 40 to 60 years.

Endari’s safety and efficacy were studied in a randomized trial of patients 5 to 58 years of age with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks.

Patients treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median three versus median four); fewer hospitalizations for sickle cell pain (median two versus median three); and fewer days in the hospital (median 6.5 days versus median 11 days).  Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6% versus 23.1%).

Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain, and chest pain.

Endari had received an orphan drug designation for this use.

Source: FDA; July 7, 2017.

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