Emmaus Life Sciences announced
that preliminary results of its Phase 3 clinical trial evaluating the safety and efficacy of L-glutamine for sickle cell anemia and sickle beta-0 thalassemia met both the primary and secondary endpoints.
The prospective, randomized, double-blind, placebo-controlled, parallel-group, multi-center clinical trial enrolled 230 adult and pediatric patients as young as five years of age, across 31 U.S. sites.
According to the press release, the initial analysis of its primary endpoint -number of occurrences of protocol-defined sickle cell crises that occurred during the 48 week trial was reduced 25% in the group receiving L-glutamine compared to those receiving placebo (p=0.008). Median frequency of hospitalizations over the 48 week study was also reduced in the L-glutamine group compared to placebo (33% reduction; p=0.018). Also, the therapy demonstrated a well-tolerated safety profile.
In the press release, Dr. Yutaka Niihara, founder and CEO of Emmaus Life Sciences said, "We are very pleased with the strength of our Phase 3 data with respect to the primary and secondary endpoints. We intend to submit a New Drug Application to the FDA in mid-2014 for marketing approval of this treatment for sickle cell disease patients."
L-glutamine has Orphan Drug Designation
in the U.S. and Europe and Fast Track designation from the FDA. Further results from the clinical trial will be released when available and are expected to be presented at a scientific meeting later this year.
About Sickle Cell Disease
Sickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, mostly African Americans. A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.
Image of a sickled red blood cell courtesy of wikimedia commons.