Continuing the Quest for Accelerated Approval

Wading through policy is no easy task.  But imagine policy as the white lines on the road, the boundaries within which FDA operates.  Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper.   Recommendation 1 encourages FDA to use Accelerated Approval for therapies intended to treat Duchenne.


Accelerated Approval is also the focus of  Emil Kakkis’ white paper (in development) which builds on this theme.  I have been involved in the working group for Emil’s paper.   On May 15, the final workshop (#5) focused on reviewing the elements for a guidance to improve access to the Accelerated Approval pathway for rare disease treatments.  We focused on surrogate endpoints – a fundamental assumption about the scientific basis of a disease, the knowledge about the drug, and its mechanism of action, which is supported by animal models and ‘reasonably likely to predict clinical benefit’.   Drs.  Marc Walton, Bob Temple, Ann Pariser participated in the meeting.  Dr. Walton focused on validation of surrogate endpoints and  Dr. Temple, expanded the discussion, providing examples of acceptable surrogates and mentioned the potential of dystrophin, as well as, muscle MRI (T2 weighted images) as possibilities.


Accelerated Approval of any drug will be good for the Duchenne community – it  will provide a clear position from FDA as to expectations and is likely to expedite other programs moving forward.  If Sarepta or any other company receives Accelerated Approval, it unlocks a door, opens the pathway, suggests a level of understanding and expertise within FDA and (cross fingers and pray) will help accelerate other potential therapies (accelerated approval, breakthrough designation, full approval)  that demonstrate safety and efficacy. 


The goal is to accelerate treatments for all individuals.  


Precision medicine – the right treatment, at the right time, for the right patient.

To slow/stop progression.

To end Duchenne.

PPMD is proud to be featured in an article from Inside Health Policy about the need for accelerated approval.

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