Adverse Reaction Deflazacort 0.9 mg/kg/day
(N=51) % at 12 weeks
Placebo (N=50)
% at 12 weeks
Cushingoid appearance 33 12
Weight increased 20 6
Increased appetite 14 2
Upper respiratory infection 12 10
Cough 12 6
Pollakiuria 12 2
Nasopharyngitis 10 6
Hirsutism 10 2
Central obesity 10 4
Erythema 8 6
Irritability 8 4
Rhinorrhea 8 0
Abdominal discomfort 6 2

The adverse reactions that were associated with deflazacort treatment discontinuation, in decreasing order of frequency, were weight increased, obesity, cataract, and sleep disorder.

Average muscle strength numerically favored EMFLAZA over placebo (Study 2)1

An additional randomized, double-blind, placebo-controlled, 104-week clinical trial evaluated deflazacort in comparison to placebo (Study 2).1

The study population consisted of 29 male children 6 to 12 years of age with a Duchenne muscular dystrophy diagnosis confirmed by the documented presence of abnormal dystrophin or a confirmed mutation of the dystrophin gene.1

The results of the analysis of the primary endpoint of average muscle strength scores in Study 2 (graded on a 0-5 scale) at 2 years were not statistically significant, possibly because of a limited number of patients remaining in the placebo arm (subjects were discontinued from the trial when they lost ambulation).1

Although not statistically controlled for multiple comparisons, average muscle strength scores at months 6 and 12, as well as the average time to loss of ambulation, numerically favored deflazacort in comparison with placebo. Many of the same adverse reactions seen in Study 1 were observed. In addition, muscle weakness, tendon disorder, and osteopenia associated with long-term steroid use were also observed.1

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1. EMFLAZA (deflazacort) [package insert]. South Plainfield, NJ: PTC Therapeutics, Inc.