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FDA approves Endari, first treatment for pediatric sickle cell disease

Wednesday, July 12, 2017

Emmaus Life Sciences announced that the FDA approved Endari (L-glutamine oral powder) to reduce the severe complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. Endari reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme that has been identified as the primary regulator of oxidation.

“The 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,” said Yutaka Niihara, M.D., MPH, chairman and chief executive officer of Emmaus Life Sciences. “Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases. 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 approximately 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. Approximately one in every 365 African American children is born with SCD and children between the ages of 2 and 7 are 400 times more likely to suffer from stroke.

Caused by a genetic mutation in the beta-chain of hemoglobin that distorts red blood cells into crescent shapes, 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 (ACS), 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 Wally Smith, M.D., Florence Neal Cooper Smith Professor of Sickle Cell Disease, Division of General Internal Medicine, Virginia Commonwealth University. “Endari has clinically shown to reduce sickle cell crises and hospitalizations, representing a significant medical advancement for patients with limited therapeutic options that have many side effects.”

FDA approval was supported by efficacy data from a 48-week randomized, double-blind, placebo-controlled, multicenter phase III clinical trial evaluating the effects of Endari, prescription grade L-glutamine, as compared to placebo on 230 adults and children with SCD. The results demonstrated that Endari reduced the frequency of sickle cell crises by 25% and hospitalizations by 33%. Additional findings showed a decrease in cumulative hospital days by 41% and lower incidence of ACS by more than 60%.

Safety was based on data from 298 patients treated with L-glutamine and 111 patients treated with placebo in the phase II and phase III studies. Endari’s safety profile was similar to placebo and well-tolerated in pediatric and adult patients. The most common adverse reactions occurring in greater than 10% of patients treated with Endari were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain and chest pain (non-cardiac).

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