FDA Approves First New Sickle Cell Drug in Almost 20 Years

The US Food and Drug Administration (FDA) on Friday granted approval to Emmaus Medical’s Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years.

Endari is approved for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. Endari is the 24th new drug approved by FDA in 2017, already two more than all of 2016.

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, said, "Until now, only one other drug was approved for patients living with this serious, debilitating condition."

Sickle cell disease is an inherited blood disorder, which according to the National Institutes of Health, affects approximately 100,000 people in the US, most often in African-Americans, Latinos and other minority groups.

FDA said safety and efficacy of Endari, which was recommended for approval by an outside panel of experts by a vote of 10-3, were studied in a randomized trial of patients aged five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial.

Development of the drug was in part supported by FDA’s Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.