ICER Researcher Questions Possible FDA Full Approval of DMD Treatment

Using Sarepta’s treatment for Duchenne muscular dystrophy (DMD) as an example, a researcher from the Institute for Clinical Review (ICER) said in a recent article that the FDA should be cautious in granting full approval to drugs that fail confirmatory tests.

The article, “The FDA and Gene Therapy for Duchenne Muscular Dystrophy,” published Wednesday in JAMA Network, describes a DMD clinical trial for Elevidys (SRP-9001) gene therapy which showed no statistically significant difference compared with placebo in boys aged 4 to 7 years.

“The change in NSAA [North Star Ambulatory Assessment] score for younger boys was greater with treatment than with placebo (4.3 vs 1.9), but statistical testing is not clearly meaningful in this post hoc analysis. Older boys had numerically worse changes with SRP-9001 than with placebo (−0.2 vs 0.5),” the article continues.

The FDA granted accelerated approval for treatment of boys aged 4 to 5 years.

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