Emmaus Life Sciences, Inc., a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies for rare and orphan diseases, has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) requesting U.S. marketing approval for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
The NDA represents the first potential treatment for pediatric patients with sickle cell disease, and the first potential new treatment in nearly 20 years for adult patients. Emmaus is requesting Priority Review of the application.
"We would like to thank the NIH, FDA and Ajinomoto Corporation for the funding of our early work at LA BioMed," said Yutaka Niihara, M.D., MPH, Chairman and CEO of Emmaus. "We are also thankful to our clinicians, employees, and partners for their efforts and to our investors for funding the work."
"We are pleased to submit our NDA during National Sickle Cell Disease Awareness Month, and following the formation of the Sickle Cell Disease Coalition spearheaded by the American Society of Hematology," Dr. Niihara added. "We hope our NDA submission will result in a change of the status quo of Sickle Cell Disease treatment."
Data from the Company's Phase 3 sickle cell disease trial demonstrated a reduction in the frequency of sickle cell crises and hospitalizations, as well as a reduction in cumulative days hospitalized, and a lower incidence of the life-threatening acute chest syndrome. The clinical trial enrolled 230 adult and pediatric patients as young as five years old, across 31 experienced sickle cell disease treatment centers in the United States. No major adverse events were attributable to the treatment.
Emmaus' sickle cell disease therapy has Orphan Drug designation in the U.S. and Europe and Fast Track designation from the FDA. Emmaus also plans to submit a marketing authorization application to the European Medicines Agency.