Sickle cell anemia is the most common and severe form of sickle cell disease, a genetic condition caused by the two copies of defective hemoglobin gene. Hemoglobin transports oxygen through the blood.
Defective hemoglobin restricts oxygen supply to the body and results in inflammation and damage to vital organs. Episodes of severe pain, called pain crisis, and anemia due to the lack healthy blood cells are characteristic of these patients.
Highly prevalent in the African-American population, more than 100, 000 Americans are affected by SCD, according to the Centers for Disease Control and Prevention.
In a Phase 3 clinical trial (NCT01179217) involving 230 patients, treatment with Endari resulted in fewer episodes of pain crisis than those receiving the placebo. Endari also significantly reduced hospitalizations, the study showed. The incidences of acute chest syndrome, a serious complication of sickle cell disease, also were markedly lower in patients receiving Endari.
With three major distribution agreements in place for Endari, this becomes the fourth largest arrangement by Emmaus for making Endari available to many more patients with sickle cell disease dealing with its complications and multiple hospital visits.
“Partnering with Cardinal demonstrates the growing demand for Endari by physicians and patients alike,” Yutaka Niihara, MD, CEO and chairman of Emmaus, said in a press release. “Teaming up with Cardinal solidifies our growing distribution network in the United States.”