February 16, 2015
Emmaus Life Sciences released results of a
phase III study of oral pharmaceutical grade
L-glutamine (PGLG) treatment for sickle cell
anemia and sickle beta-0 thalassemia. The
multi-center, double-blind clinical trial studied
PGLG for sickle cell disease (SCD) in 230 pediatric
patients as young as five years old and adults.
Study participants were randomized to receive
daily PGLG (152 patients) or placebo (78 patients)
for 48 weeks, after which treatment levels
were tapered to zero. Researchers observed
patients who received PGLG experienced fewer
painful crises (three v. four events during the
study period, a 25% reduction) and a longer
time to a pain crisis than patients receiving
placebo. Treated patients also were less likely to
be hospitalized (two v. three events during the
study period, a 33% reduction) and spent less
time in the hospital for these events (6.5 v. 11
days, a 41% reduction) than those receiving placebo.
The percentage of patients experiencing
acute chest syndrome, a severe complication of
SCD, was less than half among the PGLG group
as compared to the placebo group (11.9% v.
26.9%, or 58% fewer cases). The therapy has
Orphan Drug designation in the U.S. and Europe
and Fast Track designation from the FDA.
December 19, 2011
Novartis reported results from a phase III trial of Exjade for non-transfusion-dependent thalassemia. This one-year, randomized, double-blind, placebo-controlled pivotal study, THALASSA, enrolled 166 subjects 10 years of age and older with beta-thalassemia intermedia, alpha-thalassemia or Hemoglobin E/beta-thalassemia. The subjects were randomized to Exjade doses of 5 mg/kg/day or 10 mg/kg/day or matching placebo. Exjade at a 10 mg/kg/day starting dose significantly reduced liver iron concentration (LIC) from baseline by 3.8 mg of iron per gram of liver dry weight (Fe/g dw) compared to an increase of 0.38 mg Fe/g dw for placebo (p<0.001). The 10 mg/kg/day dose was superior to a 5 mg/kg/day dose (p≡0.009). In the 10 mg/kg arm, 49% of subjects had a LIC decrease of at least 30% from baseline versus only 2% in the placebo arm. In addition, 56% of subjects in the 10 mg/kg arm had a LIC decrease of greater than or equal to 3 mg at one year compared to 11% in the placebo arm.