Rare Disease Report

FDA Accepts Emmaus' NDA for Sickle Cell Anemia Treatment

NOVEMBER 08, 2016
The FDA has accepted Emmaus Life Sciences’ new drug application (NDA) for their orally admitted pharmaceutical grade L-glutamine (PGLG) to treat sickle cell disease. The FDA submission follows the Phase 3 trial of the drug.
The Phase 3 trial was an open-labeled randomized double blind placebo-controlled study which enrolled 230 pediatric and adult patients 5-years and older.
0.3 g of L-glutamine was administered twice a day orally to each patient for 48 weeks. The dosage was in increments of 5 grams based on weight. The upper limit for daily dose of study medication was set at 30 grams
During the Phase 3 trial, the PGLG 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.
This could be the first potential treatment for pediatric patients with sickle cell disease, if approved.
Emmaus is now awaiting notice from the FDA regarding its request for a priority review.
Last year, we talked with Yutaka Niihara, MD, MPH, chief scientific officer and founder of Emmaus Life Sciences about L-glutamine to treat patients with sickle cell disease.


About Sickle Cell Disease

Sickle cell diseas, or sickle cell anemia, 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.

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