This is the first drug approval for sickle cell disease in nearly 20 years

The U.S. Food and Drug Administration approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. This comes after nearly 20 years without a new drug approval.

Sickle cell disease, an inherited blood disorder in which the red blood cells are abnormally shaped, restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. Approximately 100,000 people in the United States have sickle cell disease, most commonly in African-American and Latinos.

Endari was studied in a randomized trial of patients ages five to 58 years old with sickle cell disease and two or more painful crises within the last year, prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo and evaluated over 48 weeks.

Patients treated with Endari experienced:

  • Fewer hospital visits for pain
  • Fewer days in the hospital
  • Fewer occurrences of acute chest syndrome

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

The FDA granted the approval of Endari to Emmaus Medical Inc.

Information from www.fda.gov.

Sickle cell anemia, 3D illustration showing blood vessel with normal and crescent shaped cells. Adobe Stock.

 

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