Sickle Cell Treatment Endari Now Available in the United States

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.

Sickle cell disease is an inherited blood disorder in which the body produces crescent shaped red blood cells that get caught in blood vessels, ultimately blocking blood flow. An altered form of hemoglobin caused by genetic mutations makes red blood cells fibrous and rigid which forms their abnormal shape. Sickled red blood cells do not last as long as normal red blood cells, causing anemia to develop in patients. Signs that a patient could have sickle cell disease begin in early childhood and include anemia, repeated infections, and periodic episodes of pain, clinically defined as sickle cell crises.

In July, the FDA approved Endari for pediatric patients 5 years of age and older who have sickle cell disease. Hydroxyurea has been the main treatment for suppressing pain episodes, making Endari the first FDA approved treatment in 20 years for adult patients and the first treatment available for pediatric patients. This approval is based on findings from a study that proved the drug to be safe and effective in dealing with pain caused by sickle cell disease.

Approximately 100,000 patients in the United States suffer from sickle cell disease.

The clinical trial included 230 patients ages 5 to 58 years who had 2 or more painful crises during the 12 months before enrollment. Patients who received Endari had fewer sickle cell crises, fewer hospitalizations, fewer days in the hospital and fewer occurrences of acute chest syndrome compared to those who took a placebo. A sickle cell crisis was described as one that resulted in the administration of a narcotic to the patient in the emergency room or the occurrence of chest syndrome, priapism, and splenic sequestration.

Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain; however, many patients in the placebo group also experienced these side effects.

“We’ve received overwhelming support and positive feedback from payers, physicians, advocacy groups and sickle cell patients, who have awaited a new treatment option for this debilitating disease for a long time,” said Yutaka Niihara, MD, MPH, chairman and CEO of Emmaus Life Sciences in a press release. “Now that Endari is available, several insurance companies have expedited their review for formulary addition to address this unmet medical need.”

Emmaus Life Sciences continues to work with patients and payers to ensure that patients with sickle cell disease receive effective treatment.

At the 59^th American Society of Hematology (ASH) Meeting and Exposition in Atlanta last December, Rare Disease Report sat down with Dr Niihara to discuss the data that led to Endari’s approval:

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