FDA approves sickle cell treatment, the first in nearly two decades

The US Food and Drug Administration (FDA) has approved Endari (L-glutamine oral powder), a treatment for patients aged five years and older with sickle cell disease, which reduces severe complications associated with the blood disorder.

“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 where red blood cells are abnormally shaped. This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage.

Endari’s safety and efficacy were analysed in a randomised trial of patients with sickle cell disease who had suffered two or more painful crises over a 12-month period before the commencement of the trial. The patient population were randomly assigned to treatment with Endari or placebo, and evaluated over a 48-week period. This study demonstrated a reduction in hospital visits for patients taking Endari and less occurrences of acute chest syndrome, compared with those on placebo.

The FDA granted Emmaus Medical with the approval for Endari.