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FDA approves Endari for sickle cell disease
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The FDA approved L-glutamine oral powder for the reduction of severe sickle cell disease–related complications in patients aged 5 years or older.
“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” Richard Pazdur, MD, 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, said in a press release. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”
A randomized trial compared the safety and efficacy of L-glutamine oral powder (Endari, Emmaus Medical) with placebo over 48 weeks in patients aged 5 to 58 years with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment.
Patients treated with L-glutamine oral powder experienced fewer median hospital visits for sickle cell crises (3 vs. 4), fewer median hospitalizations for sickle cell pain (2 vs. 3) and fewer median days in the hospital (6.5 days vs. 11 days) than the placebo arm.
Patients who received L-glutamine oral powder also experienced fewer occurrences of acute chest syndrome (8.6% vs. 23.1%).
Common side effects included constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.
The FDA previously granted orphan drug designation to L-glutamine oral powder for this indication.
In May, the Oncologic Drugs Advisory Committee voted 10-3 in favor of approval of Emmaus Medical’s new drug application for oral L-glutamine powder for chronic use in adults and children age 5 years and older with sickle cell disease.