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New drug approved for Sickle Cell disease

Cynthia E. Griffin | 10/4/2017, 3:25 p.m.

The U.S. Food and Drug Administration has approved the use of Endari (L-glutamine oral powder) for patients age 5 years and older with Sickle Cell disease to reduce severe complications associated with the blood disorder.

The FDA approved the drug for use on July 7. It is expected to be available to the public in November.

‘Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,’ said Dr. Richard Pazdur, 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.’

Sickle Cell disease is an inherited blood disorder, that typically impacts people of African descent, in which the red blood cells are abnormally shaped (in a crescent, or ‘sickle,’ shape) causing what is known as a crisis. This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. The National Institutes of Health reports that 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 approximately 40 to 60 years.

During a 48-week trial, patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered (administered or occurring elsewhere in the body other than the mouth) narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 years median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. 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 percent vs. 23.1 percent).

Endari is an amino acid indicated to reduce the acute complications of Sickle Cell disease in adult and pediatric patients 5 years of age and older.

In related news, the Sickle Cell Disease Foundation celebrated recently the one-year anniversary of its new adult clinic at Martin Luther King Jr. Outpatient Clinic, 1670 120th St., in Watts. The clinic is one of only a handful of similar facilities operating nationwide.