Rare Disease Report

Pediatric Sickle Cell Anemia Treatment Gets Go-Ahead from FDA

DECEMBER 21, 2017
Mathew Shanley
This morning, the U.S. Food and Drug Administration (FDA) granted regular approval to hydroxyurea (Siklos, Addmedica) for the treatment of pediatric patients from 2 years of age and older with sickle cell anemia who have recurrent moderate to severe painful crises.

This is the first FDA approval of the drug intended to be used as therapy in pediatric patients with sickle cell disease. It is designed to reduce the frequency of painful crises and the need for blood transfusions.

Sickle cell anemia is a genetic form of the blood disease in which there are an inadequate amount of healthy red blood cells to carry an appropriate amount of oxygen throughout the patient’s body. In sickle cell anemia, red blood cells – normally round – become rigid, sticky, and shaped like a sickle or a crescent moon. These sickle cells typically die within 2 weeks, leaving a shortage of red blood cells in the body.

Common symptoms of the condition in pediatric patients include episodic pain, swelling in the hands and feet, delayed growth, impaired vision, and frequent infections. At present, the disease affects about 100,000 U.S. residents

The approval was based on data from the ESCORT (European Sickle Cell Disease Cohort) study (NCT02516579), an open-label single-arm trial that enrolled 405 pediatric patients with sickle cell disease from 2-18 years of age. 141 patients from the cohort had not been previously treated with hydroxyurea prior to enrollment, and the drug showed promising safety and efficacy throughout the trial. After 12 months of treatment, the drug exhibited an ability to increase fetal hemoglobin in all patients, and decrease the percentage of patients who experienced at least on vaso-occlusive episode, one episode of acute chest syndrome, one hospitalization due to sickle cell disease, or one blood transfusion.

In a study published in the American Journal of Hematology this month, researchers from the St. Jude Children’s Hospital presented data from an observational study, HUSTLE (NCT00305175), that confirmed the increase of hemoglobin levels.

"…hospitalizations for the average patient fell to less than one every couple of years rather than four to six annually," said lead author Jeremie Estepp, M.D., an assistant member of the St. Jude Department of Hematology in a press release. "This frees children from the fevers, pain and other symptoms of this disease and gives them and their families more chances to enjoy childhood and adolescence."

The most common adverse reactions to the drug (incidence >10%) include infections and neutropenia. The recommended initial dose of hydroxyurea is 20 mg/kg once daily.

The FDA had previously granted priority review and orphan drug designation to hydroxyurea.

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