A Paradigm Shift at the FDA

Posted by: Christine McSherry on April 24, 2013

Day 2 at MDA: The other side of the coin - patient advocacy in drug development;
reiterate Market Outperform rating and $50 price target on Sarepta Therapeutics
based on a risk-adjusted, discounted cash flow analysis. During day 2 of the Muscular
Dystrophy Association (MDA) conference, Frank Sasinowski, attorney, former FDA
member, and board member of NORD discussed changes in the 2012 PDUFA law
that, for the first time, requires the input of patients into drug development. We have
witnessed hints of this paradigm shift from patient advocates with whom we have
interacted. Specifically in DMD, the FDA met with three mothers of boys with DMD, one
the mother of twins, and feedback that Mr. Sasinowski has received from the FDA was
that the women made a "powerful impression". In our view, this shifting FDA paradigm
is the wild card in the case for accelerated approval of eteplirsen, as the FDA may be
more receptive to a higher degree of uncertainty that they, on behalf of patients, are
willing to take on for the accelerated approval of a potential life-saving therapy. At the
conference, we also saw the 74-week data for the extension study of eteplirsen, which
is consistent with the 62-week data and, in our view, are supportive of a drug effect.
FDA evolution. The FDA is seeking input from patients, which is a paradigm shift, in
our view. To this end, the Agency will conduct panels on 16 diseases in 2013-2015
(Figure 1), focusing on important endpoints and outcomes for patients as well as what
is acceptable to them as far as risk/benefit. One of the 16 disease areas covers inborn
errors of metabolism, such as DMD. We believe the meetings between the FDA and
Sarepta and between the FDA and patient advocacy groups are representative of this
paradigm shift.
74-week eteplirsen data. Data from the 74-week time point of the open label extension
study was consistent with top-line data (Figure 2). Dr. Mendell presented these data and
stressed the lack of treatment emergent adverse events and that not a single dose was
missed or any discontinuation throughout the 74 weeks, which we believe is supportive
of a potentially differentiated safety profile."

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