Endari

Endari is an oral iron chelator medication used to treat adults and children two years of age and older with sickle cell disease who have recurrent severe anemia due to delayed diagnosis or partial usage of deferoxamine. It was approved by the US FDA on August 17, 2017.

Endari contains deferifratin, a synthetic inhibitor of the enzyme ferrireductase TRR1, which reduces the absorption of iron in the gut. This is in contrast to iron chelators such as deferoxamine that bind to and remove excess iron already in the body.

The safety and efficacy of Endari have been studied in a clinical trial involving 226 participants aged between 5 and 58 years. The results showed a significant reduction in the need for blood transfusions in patients taking Endari compared to those taking a placebo. The median duration of blood transfusions in the Endari group was 376 days, compared to 96 days in the placebo group.

Common side effects of Endari include nausea, diarrhea, abdominal pain, constipation, fatigue, and headache. More serious side effects such as hypersensitivity reactions, thrombocytopenia, and leukocytosis have been reported in some patients.

Endari is available in the form of powder packets that are mixed with oral liquid. It should be taken once a day, 30 minutes before a meal. The recommended dose of Endari is 6-10 milligrams per kilogram of body weight per day, not exceeding the daily maximum dose of 30 milligrams per day.

In 2020, Endari's manufacturer reported that sales of the medication had not increased as rapidly as expected, which raised concerns about the long-term viability of the company, Emmaus Medical.