You may or may not have heard Treat NMD has scheduled a meeting with EMEA (European Medicines Agency) or the regulatory agency for the EU. The purpose of the meeting is dialogue: discussion about potentially promising treatments for Duchenne. The discussion will cover a range of areas to include drug development, clinical trials, and outcome measures. Our champions in Congress have written to FDA, urging them to take part in this critically important conversation.
For the last many years we have been hearing about exon skipping and watching with interest as Prosensa and AVI initiated trial with compounds capable of skipping exon 51 of the Dystrophin gene. What an amazing possibility – that by skipping a single exon in the genetic sentence, the sentence would make ‘sense’, would be characterized as ‘in frame’ and with that, a shortened or truncated form of Dystrophin is expressed in muscle. For those boys whose mutation would be ‘in frame’ by skipping exon 51, this is wonderful news. But what about other mutations, where skipping a single exon would result in an in-frame mutation and a shortened version of Dystrophin?
As a community we are all very aware of the long process of drug development and while we are all want/need drugs to be safe, we also know that our sons are running out of time. The meeting in London has been developed to discuss:
- Modifying the process (the idea of seeking approval for a ‘backbone’ chemistry and less time
consuming /burdensome toxicology studies);
- Questions related to the number of patients required for clinical trials, especially for the less common exons; and
- Agreeing that status quo is improvement in a progressive debilitating condition.
Regulatory agencies will not make decisions during this meeting. We will not come away with answers. What we are hoping is that the regulatory agencies will have a better understanding of Duchenne, of this critical need, and a commitment to work with industry to accelerate the timeline.