The U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) from Emmaus Life Sciences for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease (SCD).
The NDA included data from a phase III study in which PGLG reduced the frequency of sickle cell crises and hospitalizations, as well as the cumulative number of days hospitalized and the incidence of life-threatening acute chest syndrome. The trial enrolled 230 adult and pediatric patients (≥5 years old) from 31 SCD treatment centers in the United States. No major adverse events were reported.
If the drug receives FDA approval, it will be the first new treatment approved for adults and children in nearly two decades.
The PGLG therapy previously received orphan drug designation and fast-track designation in the United States, as well as orphan medicinal product designation in the European Union.
Source: Emmaus Life Sciences press release, November 8, 2016.