Emmaus Life Sciences announced that the New England Journal of Medicine (NEJM) has published the results of its 48-week phase 3 clinical trial of Endari (L-glutamine oral powder) which supported the FDA approval in 2017 to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.
The article reports results that showed significantly fewer sickle cell crises in those receiving Endari compared to placebo by 25 percent; p=0.005 (median 3 vs. median 4) and significantly fewer hospitalizations by 33 percent; p=0.005 (median 2 vs. median 3). Additional findings showed lower cumulative days in hospital of 41 percent; p=0.02 (median 6.5 days vs. median 11 days) and a lower incidence of acute chest syndrome (ACS) by more than 60 percent; p=0.003 (13 of 152 patients [8.6%] had at least 1 ACS compared with 18 of 78 in the placebo group [23.1%]). The most common adverse reactions, occurring in greater than 10 percent, of the clinical study were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain.
“Endari is the first approved treatment for sickle cell disease in pediatric patients 5 years of age and older and the first in nearly 20 years for adults. Our hope in sharing the results of this data from the New England Journal of Medicine, a publication with worldwide reach and significance, is to aid in increasing the awareness of sickle cell disease, a lifelong hereditary blood disorder which commonly affects those of African descent, as well as those from Central and South America and people of Middle Eastern, Asian, Indian and Mediterranean descent,” said co-author Yutaka Niihara, MD, CEO and founder of Emmaus.
“Sickle cell disease affects thousands of people in the United States,” said Beverley Francis-Gibson, President and CEO of The Sickle Cell Disease Association of America, Inc. “While there is no universal cure for this life-threatening disease, patient awareness and education on treatment options remain important factors for the sickle cell community.”
The randomized, double-blind, placebo-controlled, multicenter Phase 3 trial evaluated the efficacy and safety of Endari (0.3 gram per kilogram of body weight per dose) administered twice daily by mouth, as compared with placebo. The study included patients at least 5 years of age with sickle cell anemia or sickle β0-thalassemia, with a history of two or more pain crises during the previous year. Patients who were receiving hydroxyurea at a dose that had been stable for at least 3 months before screening and who continued such therapy during the 48-week treatment period were eligible. A total of 230 patients (age range, 5 to 58 years; 53.9% female) were randomly assigned, in a 2:1 ratio, to receive L-glutamine (152 patients) or placebo (78 patients).