The Jett Foundation Fighting Duchenne Muscular Dystrophy

The Duchenne Domino Effect

April 12, 2016


Businessman Touching Domino Pieces Arranged in a Line --- Image by © Royalty-Free/Corbis

Why Eteplirsen’s Approval is Critical to the other 87 Percent of the Duchenne Population it Doesn’t Treat: The Duchenne Domino Effect

Eteplirsen only treats 13% of the Duchenne population. If FDA grants eteplirsen accelerated approval by it’s May 26th PDUFA date, most of the Duchenne community will still be without a treatment.  But with eteplirsen’s approval, comes a clearer pathway for companies interested in the Duchenne space to develop and seek approval for Duchenne therapies that treat the other genetic mutations.

This is why it is so important that you and your family attend the eteplirsen’s Advisory Committee Meeting on April 25th at the College Park Marriott in Hyattsville, MD. While eteplirsen may not treat your child, similar drugs that come after might. It is a domino effect; push eteplirsen over the finish line, and other Duchenne drugs follow. Biotech companies with oligonucleotide programs and with drugs that have alternative mechanisms of action, could be encouraged by the Duchenne Community’s fierce advocacy, and by the FDA’s recognition of eteplirsen’s safety and efficacy even in a relatively small clinical trial.

If eteplirsen is granted accelerated approval, there could be a new pathway that allows follow-on exon-skipping drugs to move faster through the regulatory system. S.2030 – Advancing Targeted Therapies for Rare Diseases Act of 2015 is a bill, sponsored by Senator Michael Bennet (Colorado), and co-sponsored by Senator Richard Burr (North Carolina), Senator Elizabeth Warren (Massachusetts), Senator Orrin Hatch (Utah), and Senator Michael Enzi (Wyoming), that would make such a pathway more likely.

S.2030 would allow the sponsor of a genetically targeted drug in the development and FDA review process to rely upon data and information previously used by that sponsor, that incorporates or utilizes the same or similar genetically targeted technology. For Duchenne, a sponsor of an approved exon-skipping drug, could potentially use data obtained from previous exon-skipping clinical trials to accelerate the approval of new exon-skipping therapies. While it would be up to the FDA to determine how much previously collected data the sponsor could use, the legislation could reduce the development and approval time of future exon-skipping drugs and allow patients access sooner than expected.

While S. 2030 is still moving through the Senate and will likely be voted on in the next few months, FDA is unlikely to use this new potential tool if they haven’t used the primary tools and authorities already granted by Congress in Section 901 of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), to approve drugs that treat an unmet medical need faster, including Accelerated Approval.

To ensure FDA uses the tools they already have, The entire Duchenne Community should call and ask their Senators to sign onto the Wicker-Klobuchar Duchenne Letter to FDA, that urges FDA to use the tools and authorities they were given in FDASIA to grant safe and efficacious potential Duchenne therapies, like eteplirsen, accelerated approval. This isn’t just a fight for one drug or for the 13% eteplirsen treats. It is a fight to encourage FDA to review Duchenne data with the tools and authorities Congress provides, to interpret and follow laws the way Congress intended, to change policy and the way that all rare disease drugs are evaluated and approved, and to push over the finish line the first Duchenne domino of approval and watch the rest tumble after it.

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