Endari for Sickle Cell Disease
The U.S. Food and Drug Administration (FDA) approved Endari in 2017 to reduce the frequency of acute SCD complications in people 5 years of age and older. This made it the first treatment approved for children with SCD and the first new treatment for adults in almost 20 years.1
What are the ingredients in Endari?
The active ingredient in Endari is glutamine.
How does it work?
We do not know exactly how Endari treats SCD. The active ingredient of Endari is an amino acid called glutamine. Sickle cells take up glutamine circulating in the blood and produce antioxidants when they break down glutamine. Antioxidants help neutralize a process called oxidative stress, which sickle cells are susceptible to. This happens when there are too many toxic chemicals that can damage proteins inside the cell.2,3
Neutralizing oxidative stress helps sickle cells become more flexible and flow better through blood vessels. This can improve oxygen delivery throughout the body, which reduces pain crises and acute chest syndrome episodes.2
What are the possible side effects?
The most common side effects of Endari include:3,4
- Abdominal pain
- Pain in the extremities, back, and chest
Other less common side effects include:3
- Blood in urine or sudden decrease in amount of urine
- Changes in skin color
- Cold hands and feet
- Dizziness and lightheadedness
- Fast heartbeat or tightness in the chest
- Rapid, shallow breathing
These are not all the possible side effects of Endari. Talk to your doctor about what to expect or if you experience any changes that worry you.
Additional things to know
Endari is not a chemotherapy, which may make it more desirable than hydroxyurea for many people. Unfortunately, Endari is much more expensive than hydroxyurea. Endari costs about $20,000 per year, compared to $1,200 for hydroxyurea. Experts have said that prices need to be lowered for most people with SCD to experience cost-effective benefits.6
There is not enough information about Endari and pregnancy to know if this drug is safe to use when pregnant or breastfeeding. The risk for birth defects and miscarriage is not known. Prior to using this drug, tell your doctor if you are pregnant or plan to become pregnant.3
Tell your doctor about any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.
For more information, read the full prescribing information of Endari.