FDA Approves New Drug Treatment for Sickle Cell Disease

On Friday, the U.S. Food and Drug Administration approved a new drug for sickle cell disease for the first time in over 20 years.

Endari, which contains amino acid L-glutamine, has been approved for use by patients, ages five and up, who have the blood disorder.

“I am hoping we are finally seeing channels opening, and that this will be the first of many new drugs to hit the market [for sickle cell disease],” according to Dr. Alexis A. Thompson, Head of the Hematology Section and Director of the Comprehensive Thalassemia Program at the Ann and Robert H. Lurie Children’s Hospital of Chicago.

Sickle cell disease is an inherited lifelong disorder in which red blood cells take on the shape of a crescent or sickle because of abnormal hemoglobin. The severity of the illness varies from person to person and typically affects African-Americans, Latinos and other minority groups.

Sickle-shaped cells are not flexible and can stick to blood vessel walls, creating a blockage that can restrict blood flow. Because of this, sufficient quantities of oxygen are unable to reach nearby tissues. These painful occurrences, called crises, often require medical attention or hospitalization. These crises can happen suddenly, without any symptoms beforehand, and can be caused by various stressors to the body, such as cold weather, dehydration or pregnancy.

“These [crises] are major disruptions to the individuals who have sickle cell disease,” Thompson notes. “Not just the severity of pain, but their ability to continue their education and to maintain their family life and a job. All are impacted if one has to be hospitalized frequently for something like pain.”

This is where Endari comes in.

The drug works by increasing the L-glutamine levels in the blood to prevent the red cells from becoming fragile and clogging blood vessels. It was studied in patients ranging from five to 58-years-old and those who had received the medication had fewer and shorter hospital visits than those on the placebo.

Common side effects associated with Endari include constipation, nausea, headache, and pains in various parts of the body, according to the FDA.

“We clearly have much room for improvement in what we can provide for people with sickle cell disease and while we are clearly very excited to see another drug reach the marketplace, we need more,” Thompson said.

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