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.