“We are pleased to partner with ADDMEDICA to provide access to an orphan drug therapy that can contribute to reducing painful crises, specifically for children with sickle cell anemia,” Éric Gervais, executive vice president of Medunik, said in a press release. “The addition of Siklos to the Medunik portfolio establishes our footprint in the United States in the area of orphan drug therapies and is the latest in a series of significant advances for our company.”
“Medunik USA is well positioned to ensure both the marketing and distribution of Siklos in the United States, thanks to their knowledge of the orphan drug commercialization process. We are pleased to count them as a partner,” said Bernard Dauvergne, executive director of ADDMEDICA.
This is the first FDA approval of hydroxyurea for use in pediatric patients with sickle cell anemia. The therapy is specifically indicated to lessen the number of painful crises and the need for blood transfusions in children ages 2 and older who have recurrent moderate to severe painful crises.
“This approval is a major milestone in treatment for all children who suffer from devastating pain and disability associated with sickle cell anemia,” said Abbey Meyers, founder and former president of the National Organization for Rare Disorders and a member of Medunik’s advisory board.
The FDA’s green light was based on data from an observational, open-label, single-arm trial, known as the European Sickle Cell Disease Cohort study, or ESCORT-HU (NCT02516579).
The study focused on the long-term safety and effectiveness of Siklos in preventing complications due to painful blockages of blood vessels in sickle cell disease patients.
Out of 405 sickle cell disease patients, ages 2-18, included in the study, 141 had not been treated with hydroxyurea prior to enrollment. Researchers reported that after 12 months of hydroxyurea-based treatment, the number of painful disease-related episodes, hospital admissions, and blood transfusions decreased. In addition, the levels of fetal hemoglobin, associated with a reduced rate of acute painful episodes and reduced disease severity, increased.
Because children’s weight often changes rapidly, Siklos allows flexible dosing to simplify adjustments made based on the child’s weight and maximum tolerated dose.