In July 2017, The U.S. Food and Drug Administration (FDA) approved the first sickle cell treatment in nearly 20 years. The treatment, Endari®, was created to help reduce complications associated with sickle cell disease (SCD). In late 2019- just over two years later- the FDA would approve two more drugs: Oxbryta™ and Adakveo®.
The following is a collection of information about the FDA approved sickle cell treatments Endari®, Oxbryta™ and Adakveo®.
Endari® was approved by the FDA for patients five and older on July 7, 2017. The treatment is used to reduce severe complications associated with SCD (e.g., acute chest syndrome).
Endari®, which is an oral L-glutamine powder, works by increasing the amount of free glutamine circulating in the blood. This glutamine is taken up by sickle cells (i.e., red blood cells that have sickled due to oxidation damage) to create anti-oxidant molecules as a product of glutamine degradation. The presence of the newly created antioxidants help neutralize the sickled red blood cells, so they can regain flexibility and properly travel through vessels without slowing blood flow and successfully carry oxygen throughout the body.
Oxbryta™ (voxelotor) was approved by the FDA for patients 12 and older on November 25, 2019. The first-of-its-kind treatment targets the fundamental cause of SCD by helping to stop the sickling of red blood cells (polymerization) and increasing the number of healthy cells properly carrying oxygen throughout the body.
By increasing the oxygen affinity for hemoglobin, Oxbryta™ stabilizes red blood cells which prevents them from becoming sickled. Healthy red blood cells, unlike those that are sickled, can maintain normal blood flow and oxygen throughout the body.
Adakveo® (crizanlizumab) was approved by the FDA for patients 16 and older on November 15, 2019. The treatment is used to help reduce the number of sickle cell crises (i.e., vaso-occlusive crises) in patients.
The drug attaches to P-selection — which contributes to the “stickiness” that occurs in sickled red blood cells — to prevent it from connecting to healthy cells. By preventing the adhesion of sickle cells to blood vessels, healthy cells can maintain normal blood flow.
Reach out to your healthcare providers to learn more about these treatments and to see if they’re right for you.
For additional information on these FDA-approved drugs, please visit dailymed.nlm.nih.gov/dailymed.
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