FDA Approves New Drug for Sickle Cell Disease


For the first time in almost 20 years, the U.S. Food and Drug Administration has approved a new drug for the treatment of sickle cell disease, a hereditary blood disorder. Endari (L-glutamine oral powder) is approved for people over the age of 5 to reduce complications of sickle cell.

In sickle cell disease, red blood cells are crescent, or "sickle," shaped. According to the National Institutes of Health (NIH), sickle cell disease affects approximately 100,000 Americans; the disorder is more common in African-Americans, Latinos, and other minority groups. In people with the disease, abnormally-shaped red blood cells restrict blood flow and the delivery of oxygen to the body’s tissues, resulting in severe pain and organ damage.

In clinical trials, patients treated with Endari experienced fewer complications, including sickle cell crises and acute chest syndrome, than patients treated with a placebo. According to Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, this drug joins hydroxyurea (approved by the FDA in 1998) as only the second medication to treat sickle cell disease complications.

Sourced from: FDA