EMFLAZA® (deflazacort) is NOW indicated for the treatment of Duchenne muscular dystrophy
in patients 2 YEARS OF AGE AND OLDER. LEARN MORE

EMFLAZA® (deflazacort) DELAYED THE LOSS OF MUSCLE STRENGTH VS PLACEBO1,2

EMFLAZA is the first and only FDA approved corticosteroid indicated to treat patients affected by Duchenne aged 5 years and older.

Below you will find how EMFLAZA performed in clinical trials. Evidence from clinical trials demonstrated that when compared to placebo, deflazacort1,2:

  • Delayed the loss of muscle strength in Duchenne patients

  • Slowed the loss of strength over time

  • Improved the ability to accomplish tasks related to activities of daily living such as standing up, walking, and climbing stairs

study design

Study 1: A randomized, double-blind, placebo- and active-controlled 52-week study (N=196)1,2

Study 1: A randomized, double-blind, placebo- and active-controlled 52-week study (N=196) 1,2 Study 1: A randomized, double-blind, placebo- and active-controlled 52-week study (N=196) 1,2

Patients were tested for muscle strength using a modified Medical Research Council (MRC) 11-point scale. A total of 18 muscle groups were tested for each patient.1,2

Primary efficacy endpoint:

Average change in muscle strength score from baseline to week 12.2

Secondary efficacy endpoint:

Average change in muscle strength score from week 12 to week 52.2

Study population:

Male children aged 5 to 15 years with an onset of weakness <5 years of age, increase in serum creatine kinase >10x ULN, and a genetic analysis of the dystrophin gene or biopsy that showed alterations in dystrophin amount or distribution in muscle.2


EMFLAZA® (deflazacort) DELAYED THE LOSS OF MUSCLE STRENGTH VS PLACEBO1

muscle strength chart muscle strength chart

EMFLAZA 0.9 mg/kg/day demonstrated statistically significant improvements in average muscle strength compared with placebo from baseline to week 12 (P=0.017).1

EMFLAZA delayed the loss of muscle strength over time (week 12 to week 52)1,2

Although not a pre-specified statistical analysis, compared with placebo, the deflazacort 0.9 mg/kg/day dose group demonstrated at week 52 the persistence of the treatment effect observed at week 12.1

Additional measures of efficacy were measured by timed function tests2

Icon of gowers sign
Time from supine
to standing
icon everyday activities
Time to climb
4 stairs
Icon of delayed walking
Time to run or
walk 30 feet
icon of wheelchair propulsion
Time to propel a
wheelchair 30 feet

Timed function test results numerically favored EMFLAZA

Based on clinical study results, deflazacort increased the ease of daily tasks, such as standing up, walking, and climbing stairs compared to placebo.1,2

Although not statistically controlled for multiple comparisons, results of several timed measures of patient function (i.e., time to stand from supine, time to climb 4 stairs, and time to walk or run 30 feet) numerically favored deflazacort 0.9 mg/kg/day at week 12, in comparison with placebo.1,2


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Understand the clinical safety profile of EMFLAZA


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1. EMFLAZA (deflazacort) [package insert]. South Plainfield, NJ: PTC Therapeutics, Inc. 2. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123-2131.