FDA Advisory Committee Votes for More Data on Duchenne Treatment


Data on ataluren, a first-in-class oral treatment for Duchenne muscular dystrophy, was considered inconclusive by a vote of 10-1.

US Food and Drug Administration’s (FDA) Peripheral and Central Nervous System advisory committee held a meeting on ataluren (translarna, PTC Therapeutics), a first-in-class oral treatment for Duchenne muscular dystrophy (DMD), in which it voted 10-1 that the data provided was inconclusive.

The committee members stated that there was a need for more data to prove the drugs’s efficacy. The FDA rejected an New Drug Application (NDA) for the drug in 2016, after which PTC reapplied in March of 2017. The drug’s potential approval to market is scheduled to be decided on by the FDA on Oct. 24.

Data from the phase 3 ACT DMD trial is being reviewed by the administration for its decisions. In the trial, the primary outcome was to measure the difference between boys treated with ataluren compared with placebo in the 6-Minute Walk Test.

ACT DMD was the largest placebo-controlled study ever conducted for DMD, consisting of 228 patients, aged 7 to 16 years old, at 53 sites across 18 countries. Patients were randomized to either 40 mg/kg of ateluren or placebo for a duration of 48 weeks. Results showed a 15-meter benefit for the ateluren group (p=0.213), but it was not found to be statistically significant.

PTC Therapeutics noted in a statement that there was, however, a statistical benefit seen in “the pre-specified patient population of 300-400 meters at baseline as measured” by the 6-Minute-Walk-Test of 47 meters (p=0.007).

The study also took into account the North Star Ambulatory Assessment (NSAA) scores of the patients, with results showing a 1.5-point difference between the two groups, in favor of the ateluren treated group, which worsened by a mean of 7.0 points compared to 8.5 points in the placebo group.

“These results show Translarna’s ability to change the course of DMD disease progression. The totality of the data from our two robust placebo-controlled studies across over 400 patients demonstrate a clinically relevant impact on patients’ lives,” Stuart W. Peltz, PhD, the chief executive officer of PTC Therapeutics, said in Oct. 2015. “We plan to submit these results to the EMA and complete our NDA submission to the FDA by the end of the year. We sincerely thank all the boys and young men, their parents, and the investigators who participated in this study for their commitment.”

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