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Promising new sickle cell drug may target complications of disease

7/13/2017, midnight

The U.S. Food and Drug Administration has approved a promising new drug developed by a Torrance pharmaceutical company to treat complications of sickle cell disease.

Emmaus Medical Inc., a subsidiary of Emmaus Life Sciences Inc., developed the drug, Endari, over a 25-year period. It’s the first and only treatment for sickle cell disease approved for pediatric patients and the first in nearly two decades for adults, according to the FDA.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Dr. Richard Pazdur of the FDA’s Center for Drug Evaluation and Research. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

Dr. Yutaka Niihara, chairman and CEO of Emmaus, said the FDA approval of Endari “is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children.”

“Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases,” he said. “We thank the FDA for its prompt review and look forward to making treatment available to patients as early as this fourth quarter.”

SCD is a rare, debilitating and lifelong hereditary blood disorder that affects roughly 100,000 patients in the U.S. and up to 25 million patients worldwide, the majority of which are of African descent as well as Latinos and other minority groups.

Nearly one in every 365 Black children is born with SCD and children between the ages of 2 and 7 are 400 times more likely to suffer from stroke.

SCD lowers oxygen levels in the blood and has an extensive impact on morbidity, mortality and quality of life. Patients often suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells block the blood vessels, resulting in excruciating pain.

Sickle cell crises can lead to organ damage, stroke, pulmonary complications, and other adverse outcomes, including acute chest syndrome, which may be potentially fatal and is the leading cause of death among people with SCD.

“A sickle cell crisis is the most common acute complication for patients and the number one cause of emergency room visits,” said Dr. Wally Smith, a sickle cell expert at Virginia Commonwealth University. “Endari has clinically shown to reduce sickle cell crises and hospitalizations.”