Sickle Cell Disease

Sickle cell disease (SCD) is a group of inherited red blood cell disorders that affects about 100,000 Americans. This disease is present in affected individuals at birth and causes the production of abnormal hemoglobin.1

Hemoglobin is a molecule in red blood cells that is responsible for delivering oxygen to organs throughout the body. Healthy red blood cells are round and flexible so they can move through even the smallest blood vessels. In someone with SCD, abnormal hemoglobin causes the red blood cells to become rigid and C-shaped, similar in appearance to a sickle.1

Sickled red blood cells die quickly, leading to a shortage of red blood cells, a condition named anemia. Also, sickled cells become stuck in the smaller blood vessels and block blood flow. The blockage of blood flow prevents tissues and organs from receiving the necessary oxygen.2

The signs and symptoms of SCD will vary from person to person because vessel blockage and insufficient oxygen transfer occurs throughout the body. Common symptoms of SCD include anemia, organ damage, ulcers, and pain crises. People with SCD are also at increased risk of life-threatening events such as acute chest syndrome.1


ENDARI is indicated to reduce the acute complications of sickle cell disease in adults and children 5 years of age and older.


The most common side effects in clinical studies were constipation, nausea, headache, pain in the stomach area, cough, pain in hands or feet, back pain, and chest pain.

Side effects that lead to a stop in treatment during the clinical study were, one case each of overactive spleen (an organ that helps filter your blood), pain in the stomach area, indigestion, burning sensation, and hot flash.

It is not known whether ENDARI is safe and effective in children with sickle cell disease younger than 5 years old.

Talk to your doctor to determine if prescription ENDARI is right for you. You may report side effects to the FDA. Visit www.fda.gov/MedWatch or call 1-800-FDA-1088. You can also call Emmaus Medical, Inc. at 1-855-725-0900.

For more information, please read the full Prescribing Information, including Instructions for Use, for ENDARI.

  1. Hematology.org. (n.d.). Sickle Cell Disease. [online] Available at: https://www.hematology.org/Patients/Anemia/Sickle-Cell.aspx [Accessed 6 Sep. 2019].
  2. Centers for Disease Control and Prevention. (2017). What is Sickle Cell Disease? | CDC. [online] Available at: https://www.cdc.gov/ncbddd/sicklecell/facts.html [Accessed 10 Sep. 2019].