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I wrote the following last month when
previewing the 48-week results:
"Positive results will show a continuation of the slower decline (stabilization) in 6MWT performance for the patients in the original 50 mg eteplirsen group. If these patients actually improve on the 6MWT at 48 weeks compared to 36 weeks, it's a home run."
Today's data were not only a home run, they were a grand slam. The 50 mg eteplirsen group actually improved walking distance over baseline. Incredible! The 6MWT results for these four boys increased from -12 meters at 36 weeks to +21 meters at 48 weeks. This is an unprecedented result for DMD patients because current treatments at best only slow the progression of disease.
Equally as stunning were the results from the group of patients who started on placebo then switched to eteplirsen after 24 weeks. I wrote this in my
"Likewise, stabilization or slowing declines in 6MWT performance for the placebo/delayed treatment group would also be positive. Remember, these patients were on placebo for 24 weeks before switching to eteplirsen. It would be great to see improvements in walking ability coming after 24 weeks of eteplirsen therapy."
Sure enough, we saw an improvement in walking ability from 36 to 48 weeks of about 10 meters. Again, remarkable because the slope of the 6MWT curve changed direction. On this morning's conference call, we learned that three of the four patients in the placebo/delayed treatment group showed "stabilization" at 48 weeks, meaning a change of +/- 10 meters on the 6MWT.
Can Sarepta make a convincing argument in front of FDA to grant early, accelerated approval of eteplirsen based on this tiny phase II study?
I say yes, although not easy even with Wednesday's stellar data. Being able to tie significant increases in dystrophin production to improved walking ability will be a big help. The FDA's new mandate to accelerate approval of new drugs for rare diseases works in Sarepta's favor, as will intense lobbying from DMD patients and their advocates. [Can you imagine the phone calls and letters FDA will receive from DMD patients after today?]