FDA to Review Novel Sickle Cell Disease Therapy
Emmaus Life Sciences have announced that the Food and Drug Administration (FDA) has accepted their New Drug Application (NDA) for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
The NDA is based on results from Emmaus's Phase 3 trial, which included 230 adult and pediatric patients (as young as 5-years-old). The trial demonstrated a reduction in the frequency of sickle cell crises and hospitalizations, as well as a reduction of hospitalized days and lower incidence of life-threatening acute chest syndrome.
The FDA previously granted Orphan Drug and Fast Track designations to the Company's orally administered pharmaceutical grade L-glutamine.
If approved, the treatment will become the first for pediatric patients with sickle cell disease, and the first potential new treatment in nearly 20 years for adult patients.
For more information visit emmauslifesciences.com.