#MDA2022 – Emflaza Outperforms Prednisone in DMD Analyses

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The corticosteroid Emflaza outperforms prednisone at slowing disease progression in people with Duchenne muscular dystrophy (DMD), according to new research presented at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting being held this week in Nashville, Tennessee.

The research was funded by PTC Therapeutics, which markets Emflaza.

Corticosteroids are a class of anti-inflammatory medications that work by mimicking a naturally occurring hormone called cortisol, and are a part of standard care for DMD. Prednisone is a well-established corticosteroid widely used for this purpose. Emflaza (deflazacort) is a more recently developed corticosteroid, first authorized for use in DMD patients in the U.S. in 2017.

Using data from two natural history studies, called PRO-DMD-01 (NCT01753804) and CINRG, researchers compared outcomes for 175 boys with DMD treated with prednisone against 288 boys given Emflaza. In both groups, the mean age at diagnosis was around 5 years, and the mean age was 9–10 at the study’s start.

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The researchers compared the time it took the boys on either treatment to reach certain markers of worsened motor function — for example, taking longer or being unable to stand up from lying down on the floor, being unable to climb four stairs, or being unable to walk. Findings were presented in a poster titled, “Associations Between Daily Deflazacort or Prednisone and Ages at Disease Progression Milestones Among Patients with Duchenne Muscular Dystrophy (DMD).”

On average, the boys in this study were on steroid treatment for nearly three years.

Results showed that those treated with prednisone tended to hit all these markers at significantly younger ages than those given Emflaza.

“Use of daily [Emflaza] was associated with delayed progression of multiple ambulatory milestones relative to daily prednisone in patients with DMD,” the researchers concluded.

In another poster, titled, “Associations Between Steroid Treatment and Clinical Outcomes Among Non-amublatory Patients with Duchenne Muscular Dystrophy (DMD),” researchers analyzed data from PRO-DMD-01, focusing only on those who were unable to walk.

Data for 29 children treated with prednisone, 40 with Emflaza, and 17 who did not receive any corticosteroid treatment were analyzed. The average age for all groups at the study’s start was about 13.

Results of Performance of Upper Limb, a measure of upper limb function, showed that scores declined significantly more slowly for those given Emflaza than prednisone, and both corticosteroids led to better outcomes compared to no treatment.

Outcomes were consistently better in measures of heart and lung function for those treated with Emflaza or prednisone compared with those who received no corticosteroids, but there was no clear difference between the two corticosteroids. Both also reduced the risk of being unable to raise the hands to the mouth or turn over in bed, but there was no difference between them.

“Steroid use after loss of ambulation was associated with delayed progression of important pulmonary, cardiac and functional deficits in DMD,” the researchers said, emphasizing the importance of continuing steroid treatment even after patients are unable to walk.

Another similar study was conducted using data from children given a placebo (in addition to standard-of-care corticosteroids) in four clinical trials that tested experimental DMD treatments (NCT01826487, NCT01865084, NCT00592553, and NCT01254019). The results were detailed in the poster, “Associations Between Deflazacort vs. Prednisone/Prednisolone and Disease Progression Markers in Subgroups of Patients with Duchenne Muscular Dystrophy.

The study included 127 DMD boys treated with daily Emflaza and 104 given prednisone, with a mean age of 9 at the beginning of the study.

Outcomes on a number of measures were significantly better after about a year of treatment among those given daily Emflaza instead of daily prednisone. These included the distance the boys could walk in six minutes, the time it took to climb up or down four steps, and the North Star Ambulatory Assessment score, a measure of functional motor abilities in DMD patients able to walk.

“Benefits of daily [Emflaza] compared to daily prednisone for preserving ambulatory function in DMD were most evident among patients who were older, had been on steroids longer [more than three years], or were at a more progressed disease stage. These results add to the evidence for a potential cumulative benefit of [Emflaza] versus prednisone,” the scientists concluded.


Note: The Muscular Dystrophy News Today team is providing coverage of the 2022 MDA Clinical and Scientific Conference March 13–16. Go here to see the latest stories from the conference.