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Update: At the advisory committee meeting on Nov. 24, most panel members voiced doubts that the treatment was effective. A final FDA decision on drisapersen is expected by Dec. 27.

The fate of a drug to combat Duchenne muscular dystrophy may be in doubt after Food and Drug Administration staff raised serious concerns about the safety and effectiveness of the treatment. The assessment was contained in documents released on Friday in advance of an FDA advisory panel meeting next week to review BioMarin Pharmaceutical’s drisapersen, one of two new experimental medicines for DMD slated for regulatory review.


Listen to the Signal podcast: For boys with Duchenne, and two drug companies, a moment of shared hope

The FDA reviewers cited “life-threatening” side effects, such as a low blood platelet count and severe kidney damage. They also noted a lack of “substantial evidence” of effectiveness, pointing to a failed late-stage study and an inability to substantiate findings from a mid-stage study showing the mobility of boys taking the drug improved after a six-minute walking test. And they argued that the BioMarin drug did not seem to boost levels of dystrophin, the protein that is missing in boys with DMD and which is meant to be restored by the treatment.

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