20-year Approval Hiatus Broken, Not Once, But Twice!

A couple of days before Christmas, the FDA granted a regular approval to the drug, hydroxyurea (Siklos) for treatment of pediatric sickle cell anemia.

Sickle cell anemia is a chronic disease that creates stiff red blood cells that get caught along the vessel walls. In turn, these blockages halt the flow of oxygen, causing pain, infections, or even organ damage. Currently, transfusions are used to help manage the disease, but these are invasive and difficult for patients, especially children.

Hydroxyurea is used to help decrease the incidence of moderate-severely painful crises in patients and the need for transfusions in pediatric patients who are 2 years of age and older. It works by increasing hemoglobin F levels, which was demonstrated during a huge Cohort study of 405 pediatric patients. Furthermore, these results were met with adverse reactions in fewer than 10% of patients treated with the drug.
In fact, hydroxyurea is actually the first FDA approval for a medication specifically targeting pediatric patients with sickle cell anemia! Needless to say, this is great news for a community desperately seeking better treatment.
It is shaping up to be a fantastic year for the sickle cell anemia community. Earlier this year, the FDA also approved L-glutamine oral powder (Endari) for treatment, as well.
This was the first sickle cell drug approved in 20 years and definitely something to celebrate from 2017!

To learn more about the latest approval of hydroxyurea, click here!


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