Addmedica has announced that the US Food and Drug Administration (FDA) has approved Siklos (hydroxyurea tablets) in pediatric patients, two years of age and older, suffering from sickle cell anemia.
Approval was obtained under a priority review and orphan drug designation.
Siklos offers dosing options to facilitate adjustment based on the patient’s weight to obtain the best efficacy/tolerance ratio for each child throughout the growth years.
FDA-approval of Siklos was granted based on efficacy and safety data collected in a cohort study, Escort-HU, which included several hundred child and adult patients treated with Siklos in Europe.
“FDA-approval of Siklos is a major breakthrough in the treatment of American children with sickle cell anemia. European patients have been benefitting from Siklos, the only form of hydroxyurea indicated in pediatric care in the world, since 2007,” François Anger, President of Addmedica. “This successful outcome can also be attributed to the patients who agreed to participate in the Escort-HU study and the physicians who allowed it to happen. With the new FDA-approval, ADDMEDICA consolidates its leadership in the treatment of sickle cell anemia by continuing to invest in the development and availability of this essential therapeutic product in new countries.”
Siklos will be marketed in the United States by Medunik USA Inc.
Sickle cell anemia is a particularly disabling disease, whose most common acute manifestations are painful vaso-occlusive crises in the bones, lungs (acute chest syndrome) and most vascularized organs, and strokes. It may be life-threatening, especially in the case of acute chest syndrome.
The disease is caused by abnormal hemoglobin molecules causing red blood cells to become stiff and sickle-shaped under certain conditions whence the name of sickle-cell anemia.
These sickled red blood cells tend to pile together, creating clots, and blocking the flow of blood through vessels and thus causing vaso-occlusive crises.
The disease is accompanied by varying degrees of anemia and chronic complications may appear that affect various organs, e.g. hepatic or renal impairment and retarded growth.
The clinical expression of the disease and its evolution are highly variable among individuals.
Painful vaso-occlusive crises are the most common cause of hospitalization in sickle cell anemia patients, and therefore represent a very high cost for society.
Hydroxyurea is currently recognized as the reference drug treatment for this disease.
Approximately 100,000 persons suffer from sickle cell anemia in the USA compared with approximately 60,000 in Europe. In both cases, it is considered to be a rare disease (less than 5 cases per 10,000 inhabitants).