WIKIMEDIA, FDAAn advisory panel to the US Food and Drug Administration (FDA) convened Tuesday (November 24) to evaluate the strength of evidence for drisapersen, a drug for the often fatal muscle disease Duchenne muscular dystrophy (DMD). Drisapersen, produced by BioMarin Pharmaceutical, has been tested in three placebo-controlled clinical trials, but the panel concluded none had shown clear evidence that the drug was helping. By a vote of 15-2, the panel found that the largest trial, which had measured the distance patients walked in six minutes, didn’t show significant improvement in those receiving drisapersen. And most panelists even voted that two of the studies had actually “weakened” the drisapersen’s case for approval, Xconomy reported. “Efficacy has not been established,” Glen Nuckolls of the National Institutes of Health told The Wall Street Journal.
The panel did leave open the possibility that drisapersen could help some children, but it did not vote on whether members through drisapersen should be approved. “We want to gain input from the panel and will engage in substantive discussions today and discuss strength of evidence,” FDA spokesperson Sandy Walsh wrote in an email to Xconomy. The FDA has already raised concerns about the drug’s safety, noting that it has had adverse effects on the kidneys, skin, and blood vessels. “[E]ven in the context of an invariably disabling and fatal disease such as DMD, the safety profile of drisapersen is concerning,” the agency wrote in a written report, according to The Wall Street Journal.
An approval decision is highly anticipated, as patients and parents are ...
.jpg){kind=link}