FDA approves Endari (L-glutamine oral powder) in sickle cell disease- Emmaus Life Sciences

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. 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 percent and hospitalizations by 33 percent. Additional findings showed a decrease in cumulative hospital days by 41 percent and lower incidence of ACS by more than 60 percent.

Safety was based on data from 298 patients treated with L-glutamine and 111 patients treated with placebo in the Phase 2 and Phase 3 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 percent of patients treated with Endari were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain (non-cardiac).