New Sickle Cell Treatment Approved

This is the first treatment for the disorder in 20 years.

Intended for all patients age 5 and older, the FDA announced that it has approved Endari (L-glutamine oral powder) to treat sickle cell disease and reduce the severe complications that often result from the blood disorder.

An inherited disorder, the FDA explained sickle cell disease is identified by abnormal, crescent or “sickle” shaped red blood cells that in effect, limits blood flow and restricts oxygen delivery to body tissues. Symptoms include intense pain and organ damage; according to the National Institutes of Health affect more than 100,000 people in the U.S. The average life expectancy of patients afflicted with the disease, said the FDA, is approximately 40 to 60 years of age.

Richard Pazdur, director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research explained, "Endari is the first treatment approved for patients with sickle cell disease in almost 20 years. Until now, only one other drug was approved for patients living with this serious, debilitating condition."

Endari’s safety and efficacy were, said the FDA, studied in a randomized trial of patients ranging in age from 5 to 58 who had experienced two or more painful crises within 12 months prior to enrolling in the trial. Per common procedure, patients were randomly selected and treated either by placebo or Endari for 48 weeks. “Patients,” said FDA, “who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac.” On average, explained FDA, compared to patients treated by placebo the treatment resulted in fewer pain-related hospitalizations and shorter stays if hospitalized for pain. “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,” said FDA.

Endari received orphan drug designation for this indication; the FDA and reviewed Emmaus Life Sciences New Drug Application for the drug last May. Development of Endari, according to the FDA, was in part supported by the agency’s Orphan Products Grants program that funds grants for clinical studies.

 

Cynthia A. Challener, Ph.D.

Dr. Challener is an established industry editor and technical writing expert in the areas of chemistry and pharmaceuticals. She writes for various corporations and associations, as well as marketing agencies and research organizations, including That’s Nice and Nice Insight.

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