Rare Disease Report

FDA Advisory Committee Gives Thumbs Up for L-glutamine for Sickle Cell Disease

MAY 24, 2017
James Radke
The U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee voted 10-to-3 that the overall Benefit-Risk profile of Endari (L-glutamine) for treating patients with sickle cell disease is favorable.
The FDA is set to make its final decision on the drug by July 7, 2017.
If approved, Endari would be the first FDA-approved treatment for pediatric patients with sickle cell disease, and the first new treatment for adult patients in almost 2 decades.
Sickle cell disease is a rare genetic condition that affects 90,000-to-100,000 Americans, mostly African Americans.  A sickle cell disease patient 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.
Patients with sickle cell disease often suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells occlude blood vessels. Sickle cell crises cause excruciating pain may lead to organ damage, stroke, pulmonary complications, skin ulceration, infection and a variety of other adverse outcomes.

Efficacy and Safety

The Committee focused on the phase 3 clinical trial in which patients with sickle cell or sickle ß0 -thalassemia aged 5 years and older were randomized (2:1) to receive oral L-glutamine 0.3 mg/kg/day or placebo for 48 weeks.
The primary outcome measure was the frequency of sickle cell crises and, after 48 weeks, the median number of sickle cell crisis events was 3 in the L-glutamine arm and 4 in the placebo arm (P = .0052).
The FDA documented that while the difference was statistically different, the efficacy data was complicated by differences in discontinuation rates observed during the course of the study. While that was a concern, the FDA documented that their own exploration of the data favored L-glutamine over placebo in reducing the rates of crises.
The safety review revealed that the most common adverse reactions (incidence ≥ 10% and greater than placebo) were constipation, nausea, headache, cough, pain in extremity, back pain, chest pain, and abdominal pain.
RDR has spoken with Dr Niihara about L-glutamine to treat sickle cell disease on several occasions. Below are 2 clips from those interviews:


In January, Canada-based Generex Biotechnology announced its purchase of a 51% share of Emmaus for $225 million.

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