Absolutely on-point article regarding the recent approval of Sarepta Pharmaceutical's DMD drug Exondys 51 (eteplirsen). There is no doubt that DMD is a horrible disease and any potential treatment of that disease must be expedited. However, the efficacy of Sarepta's drug was questioned by the FDA's own medical panel.
Amid prior mistakes and botched FDA submissions, Sarepta launched a public relations coup, with many parents of children with DMD traveling to Washington DC to lobby the FDA directly to approve etepirsen. Ultimately, the FDA's head of drug review division approved the medication, overruling the FDA's own medical panel amid some bitter intra-agency dissent.
The FDA has struggled recently with bureaucratic processes that would bring badly-needed drugs to the market faster, while trying to maintain the same strict efficacy requirements that have been in place for decades.
Amid pressing news in other sectors, this approval stands out as very unusual and quite news worthy. Statnews does an excellent job covering the topic in the article hyperlinked below.
https://www.statnews.com/pharmalot/2016/09/19/sarepta-fda-duchenne-behind-the-decision/
Amid prior mistakes and botched FDA submissions, Sarepta launched a public relations coup, with many parents of children with DMD traveling to Washington DC to lobby the FDA directly to approve etepirsen. Ultimately, the FDA's head of drug review division approved the medication, overruling the FDA's own medical panel amid some bitter intra-agency dissent.
The FDA has struggled recently with bureaucratic processes that would bring badly-needed drugs to the market faster, while trying to maintain the same strict efficacy requirements that have been in place for decades.
Amid pressing news in other sectors, this approval stands out as very unusual and quite news worthy. Statnews does an excellent job covering the topic in the article hyperlinked below.
https://www.statnews.com/pharmalot/2016/09/19/sarepta-fda-duchenne-behind-the-decision/
