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#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

April 20, 2018

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials.

The new findings follow a reassessment of pooled data obtained in the placebo group of the Study 007 Phase 2b (NCT00592553) and ACT DMD Phase 3 (NCT01826487) clinical trials. The aim of the data re-analysis was to evaluate the effect of corticosteroids on DMD disease progression, since all participants in the study were being treated with corticosteroids.

Results of the pooled data analysis are scheduled to be presented at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, April 21-27. They will be shown in a poster titled “Meta-Analysis of Deflazacort vs Prednisone/Prednisolone in Patients with Duchenne Muscular Dystrophy,” as part of the session titled Child Neurology and Developmental Neurology: Neuromuscular Disease.

The trials were initially designed to demonstrate the efficacy of PTC Therapeutics’ investigative drug Translarna (ataluren). About 400 boys with DMD were included in the studies and were randomized to receive placebo or Translarna.

The participants who were enrolled in the placebo groups received corticosteroids therapy for 48 weeks before the trial initiated. Sixty-four boys were treated with Emflaza and 82 with standard care based on prednisone or prednisolone. In what is known as a post-hoc analysis, the researchers re-analyzed and compared the data collected from these patients that related to motor function, as measured by the 6 minute walk test (6MWT).

They found that DMD patients who received Emflaza for 48 weeks were able to walk longer distances than those treated with standard care corticosteroids. The estimated mean difference was of 34.1 meters in favor of the Emflaza-treated group.

Deflazacort [Emflaza] appeared to be more effective than prednisone/prednisolone in delaying progression of DMD,” the researchers wrote.

Results of a prior post-hoc analysis of ACT DMD trial data had already showed a similar improved response, with Emflaza-treated patients being able to walk 31.6 meters more than the other boys. Also, Emflaza delayed loss of ambulation, or walking ability, in boys older than seven by a mean time of 3.8 years compared to prednisone or prednisolone.

The most common adverse events in both treatment groups affecting more than 10 percent of the participants were vomiting, headache, inflammation of the nasal cavity and pharynx (nasopharyngitis), fall, diarrhea, upper abdominal pain, cough, pain in extremity, and fever.