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I haven't written about
Sarepta Therapeutics(SRPT - Get Report) lately mainly because there hasn't been much change to the story. I'm as bullish as I've ever been about the efficacy and safety of eteplirsen and the FDA agreeing to an early approval filing. I still believe the FDA's affirmative decision to allow Sarepta to file based on data from the current, small phase II study (when it happens) is tantamount to an approval. The eteplirsen data have strengthened over time. Parents and advocates working on behalf of the Duchenne muscular dystrophy kids have been very effective at lobbying FDA and Congress, pushing for early approval of eteplirsen.
FDA, in turn, has been incredibly responsive, even solicitous, to the DMD community. The agency's top officials -- Margaret Hamburg, Janet Woodcock and Bob Temple -- are all personally involved in the eteplirsen review. [Yes, eteplirsen is already under review by FDA even without an "official" filing. They're not swapping summer grilling recipes at all these face-to-face meetings.]
I checked in by phone recently with Jenn McNary, advocate and mother to two children, Max and Austin, with DMD. As most Sarepta followers know, McNary has been very involved in the fight for eteplirsen's early approval. Max is a patient in Sarepta's phase II study and has benefitted tremendously from eteplirsen. Austin, wheelchair bound, has not been able to get eteplirsen, unfortunately.
"There is no way the FDA is giving us this level of feedback and access just to shut us down," said McNary.
Is McNary worried at all about FDA telling Sarepta that another, larger study is required before seeking eteplirsen's approval?
"It's not going to happen," she said, without hesitation.
Sarepta bulls are accused by the bears of being overly emotional and not looking objectively at the paucity (and problems) with the eteplirsen data. There's some truth to the charge. Emotion and anecdotal evidence have factored into my bullishness, I readily admit. But I've met Max and Austin personally and it's very hard to be an eteplirsen skeptic when you watch Max glide across a room with almost no discernible limp to fetch a plate of food for his brother Austin, confined to a wheelchair.