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 nearly 20 years.1

Endari reduces the frequency of sickle cell crises and hospital visits. Endari can be taken alone or with hydroxyurea, but it is more costly than hydroxyurea.

What are the ingredients in Endari?

The active ingredient in Endari is glutamine.

How does Endari 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 of Endari?

The most common side effects of Endari include:3,4

  • Constipation
  • Nausea
  • Headache
  • Cough
  • 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 when taking Endari. You also should call your doctor if you have any changes that concern you when taking Endari.

Things to know about Endari

Endari is not a chemotherapy drug. This may make it more desirable than hydroxyurea for many people. Unfortunately, Endari is much more expensive than 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. Before using this drug, tell your doctor if you are pregnant or plan to become pregnant.3

Before beginning treatment for SCD, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you take. This includes over-the-counter drugs.

For more information, read the full prescribing information of Endari.

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Written by: Matthew Zajac | Last reviewed: August 2021