Rare Disease Report

FDA Approves Hydroxyurea Tablets for Pediatric Sickle Cell Anemia

MARCH 15, 2018
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has approved hydroxycarbamide/hydroxyurea tablets (Siklos) in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

The therapy is the first and only hydroxyurea-based treatment for the indication.

The approval was based on positive safety and efficacy data collected in the Escort-HU study (NCT02516579). It had previously been granted orphan drug designation and priority review.

In the trial, the antimetabolite was used in current practices for the prevention of symptomatic vaso-occlusive complications in several hundred pediatric and adult patients with sickle cell anemia. After 12 months of treatment, the drug demonstrated the ability to increase fetal hemoglobin in all patients, while decreasing 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.

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

“FDA-approval of Siklos is a major breakthrough in the treatment of American children with sickle cell anemia,” said François Anger, President of Addmedica in a press release. “European patients have been benefitting from Siklos, the only form of hydroxyurea indicated in pediatric care in the world, since 2007. 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.”

The drug will be available for oral use as a 100 mg or 1,000 mg film-coated tablet. In the United States, hydroxycarbamide will be marketed by the Pennsylvania-based Medunik USA, Inc. Its initial recommended dosing is at 20 mh/kg once daily based on the patient’s actual or ideal weight, whichever is less.

The most frequent adverse reactions to the drug (incidence >10%) include infections and neutropenia.

“We’re pleased to partner with Addmedica to distribute this orphan drug which is recognized as the reference drug treatment in sickle cell anaemia. It will improve the health and quality of life of hundreds of children with the disease in the United States,” said Éric Gervais, Executive Vice-President of Medunik. “The launch of Siklos in the next few months establishes our footprint in the United States in orphan drug therapies and is the latest in a series of significant milestones for the company.”

Typical symptoms of the condition in pediatric patients include episodic pain, swelling in the hands and feet, delayed growth, impaired vision, and frequent infections. The disease currently affects an estimated 100,000 U.S. residents

For more breaking news pertaining to the rare disease community, follow Rare Disease Report on Facebook and Twitter.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.