Patients with sickle cell disease will soon have access to a new treatment following the US Food and Drug Administration's (FDA) approval of Endari (L-glutamine oral powder). The drug was cleared for use in reducing some of the serious complications often experienced by patients aged five years of age and older who have sickle cell disease. 

Emmaus Medical Inc. received Orphan Drug designation for Endari for use treating sickle cell disease. The announcement comes the week after the FDA committed to clearing the backlog of Orphan Drug requests within a 90-day timeframe as part of its Orphan Drug Modernization Plan. Orphan Drug designation may be granted to medication used to treat conditions affecting less than 200,000 people in the United States, and it comes with a number of incentives to encourage development of drugs to treat these rare diseases. 

The importance of the latest FDA approval was highlighted by 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, who said: "Endari is the first treatment approved for patients with sickle cell disease in almost 20 years… Until now, only one other drug was approved for patients living with this serious, debilitating condition."

About Sickle Cell Disease

Sickle cell disease in an inherited blood disorder in which patients have abnormally shaped red blood cells which die early, causing a constant shortage of red blood cells. National Institutes of Health figures indicate it affects around 100,000 people in the US, with the average life expectancy of patients usually between 40 and 60 years. The only known cure is a bone marrow or stem cell transplant; risky and in some cases life-threatening procedures with the potential for serous side effects. 

Consequently, treatment usually focuses on avoiding crises, managing pain and symptoms, and preventing complications. A variety of medication is used to manage and treat sickle cell disease, including antibiotics such as penicillin V potassium, antimetabolites like Hydrea (hydroxyurea), and pain relieving drugs.

About Endari

Endari's safety and efficacy was shown in a randomized trial of patients with sickle cell disease between the ages of five and 58, all of whom had suffered two or more painful crises in the year before they enrolled in the trial. The study showed those who were administered Endari rather than a placebo spent fewer days in hospitals and had fewer hospital visits for pain and sickle cell crises. 

Wally Smith, MD, Florence Neal Cooper Smith Professor of Sickle Cell Disease in the Division of General Internal Medicine at Virginia Commonwealth University, stated: "Endari has clinically shown to reduce sickle cell crises and hospitalizations, representing a significant medical advancement for patients with limited therapeutic options that have many side effects."

Among the most common side effects of the medication were nausea, constipation, abdominal pain, cough, pain in the extremities, back pain and chest pain.