Emmaus Life Sciences announced that the Food and Drug Administration (FDA) has accepted for review the New Drug Application for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
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 U.S. No major adverse events were attributable to the treatment. If approved, this 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.
The company is awaiting notice from the FDA regarding its request for a priority review. Emmaus' sickle cell disease therapy has Orphan Drug designation in the U.S., Orphan Medicinal Product designation in the EU and Fast Track designation from the FDA. Emmaus also plans to submit a marketing authorization application to the European Medicines Agency.