Top-Line Results for Sickle Cell Disease Meet Trial Endpoints
Emmaus Life Sciences announced that preliminary results from its Phase 3 clinical trial evaluating the safety and efficacy of oral L-glutamine for sickle cell anemia and sickle beta-0 thalassemia met both primary and secondary endpoints.
The prospective, randomized, double-blind placebo-controlled, parallel-group, multicenter clinical trial enrolled 230 patients >5 years old that had been diagnosed with sickle cell anemia or sickle beta-0 thalassemia and had >2 documented episodes of sickle cell crisis within 12 months of the screening visit. Data from initial analysis showed a statistically significant 25% reduction in the median frequency of sickle cell crises (P=0.008) over a 48-week period, meeting the study's primary endpoint.
Regarding the study's secondary endpoint, data showed a statistically significant 33% reduction in the median frequency of hospitalizations (P=0.018) over a 48-week time period.
The company plans to submit an NDA to the FDA in mid-2014. The therapy has been granted both Orphan Drug and Fast Track designations from the FDA.
For more information call (877) 420-6493 or visit Emmausmedical.com.