FDA Approves New Option for Sickle Cell Disease

The US Food and Drug Administration has recently approved Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.

Endari is administered orally twice a day, and can be mixed with 8 ounces of a cold or room temperature beverage or can be consumed after 4 to 6 ounces of food is ingested. The recommended dose ranges from 10 grams to 30 grams per day based on the body weight of the patient.

Approval of Endari was based on data from a randomized clinical trial that included 230 patients from 5 to 58 years of age with sickle cell anemia or sickle β⁰-thalassemia who were randomly assigned to receive Endari or placebo. During the 48 weeks of treatment, patients who received Endari had a median of 3 sickle cell crises compared with patients in the placebo group who had a median of 4 sickle cell crises. Those who received Endari experienced fewer hospitalizations associated with sickle cell pain, spent fewer days in the hospital, and had a lower incidence of acute chest syndrome.

Common adverse reactions associated with Endari included constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain. In addition, 5 patients (2.7%) had to discontinue treat due to adverse reactions, including hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash.

—Melissa Weiss

Reference:

FDA approved L-glutamine powder for the treatment of sickle cell disease [press release]. FDA. July 7, 2017. https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm566097.htm. Accessed July 7, 2017.