Rare Daily Staff

The Food and Drug Administration approved Addmedica’s Siklos to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients 2 years of age and older with sickle cell anemia, a rare genetic disease that causes an insufficient number of healthy red blood cells to carry oxygen throughout the body.

The agency approved the drug based on data from an open-label single-arm trial of 405 pediatric patients with sickle cell disease from 2-18 years of age, of which 141 had not been previously treated with Siklos prior to enrollment.

The study showed that Siklos use resulted in an increase in hemoglobin. The percentage of patients with at least one vaso-occlusive episode, one episode of acute chest syndrome, one hospitalization due to sickle cell disease, or one blood transfusion decreased after 12 months of treatment.

The most common adverse reactions to Siklos included infections and neutropenia, a low white cell count.

The FDA granted priority review and Orphan Drug designation to Addmedica for Siklos.

December 21, 2017

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