Advancing Targeted Therapies for Rare Diseases Act Goes Before HELP Committee

For nearly two years, PPMD has been working with Congressional champions and industry partners to help lead an effort to streamline the regulatory pathways and review processes for targeted therapeutics (which would include such things as ‘follow on exons’) by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy. These efforts have yielded a provision in the House of Representatives landmark bill HR 6, the 21st Century Cures Act which was approved last summer.


In the Senate, the approach was to introduce a stand-alone bill in the hopes that it would create enough momentum to become a part of the Senate companion to 21st Century Cures (the Innovation package). The stand-alone bill in the Senate bill is called the Advancing Targeted Therapies for Rare Diseases Act of 2015 – now 2016 - (S. 2030). The plan worked – with one caveat. The vehicle that was intended for the provision/bill changed as the Innovation package has not moved as a complete entity as anticipated. Today, the HELP Committee was scheduled to mark up several bills that are intended to advance biomedical innovation, among them – S.2030. 

As our community knows better than anyone, the timing could not be more appropriate and necessary. With two Food and Drug Administration (FDA) Advisory Committee meetings anticipated this year, and a robust therapeutic pipeline with other applications expected over the coming months, this legislation comes at a crucial time for the Duchenne community. With one of the anticipated product reviews for first quarter 2016 being for an exon skipping therapy (Sarepta’s PDUFA date was just moved to May 26, 2016), with follow-on exon therapies and their regulatory pathway directly impacted by this bill, the relevance of this bill is timely and on target for Duchenne.

The HELP Committee mark-up is a tremendous win for our community and we will continue to keep you posted on ways you can help urge your Members of Congress to keep momentum going.

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Comment by Jay Griffin on February 19, 2016 at 7:43am
Me too...
Comment by Shelly on February 19, 2016 at 7:40am
Thanks Jay!
I'm also working on it but obviously HT100 incident has totally shaken me up so thought of taking as many advises as I can....
Comment by Jay Griffin on February 19, 2016 at 7:33am
Shelly I.M me and we can chat
Comment by Jay Griffin on February 19, 2016 at 7:31am
Shelly, I don't think there are trials yet for skipping 45. I am not sure about the trial you are participating in. Doing our due diligence about these trials can be difficult. I often google the trial to get as much understanding about who is doing it, what they are expected to accomplish, criteria for entering the trial. I would check you tube to see if there are any webinars regarding it. I often reach out to my Dr. to see if he is familiar with the trial. There is ALWAYS risk but as a parent we want to mitigate those risk. We hope the trial will benefit our sons while info is gathered regarding safety etc.
Comment by Shelly on February 19, 2016 at 6:35am
I'm planning to participate in BMS ANTIMYOSTATIN.. Can anybody suggest me whether I am on a right track or not.. Is there any exon skipping for my son who is missing exon 45 in his dystrophin gene and needs exon 44 drug in upcoming months.. Please help!
Comment by Jay Griffin on February 18, 2016 at 8:17pm
I have a question about this posting. "A robust therapeutic pipeline with other applications expected over the coming months" How many are expected? What is the timetable? What are their area of focus?
Comment by Jay Griffin on February 10, 2016 at 7:00pm
We appreciate your efforts very much
Comment by Alpa Khushalani on February 10, 2016 at 2:29pm

Many congratulations to all of us. Thank you PPMD. We are one of the families that are waiting on trials for follow on exons and truly appreciate this effort.

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