On Friday, Pfizer presented the first results in humans for its gene therapy to treat Duchenne muscular dystrophy. Pfizer shares were flat on the announcement, but shares in Sarepta Therapeutics, which is developing its own treatment, increased 18%. STAT senior writers Adam Feuerstein and Matthew Herper sat down with some highly caffeinated beverages this morning to discuss the results.
Adam: Hey, Matt, the consensus seems to be that Sarepta Therapeutics has a more effective and safer gene therapy than Pfizer. That’s the big takeaway from the Pfizer presentation on Friday. How do you feel about that?
I think your comment that Pfizer deserves to be criticized for using a ‘new’ dystrophin detection assay is misplaced. First, it isn’t that new (first presented 2-3 years ago), it is a significant advance in measuring dystrophin (greater sensitivity, much wider dynamic range), and according to the slides Pfizer presented, the FDA reviewed it and gave a thumbs up.
It is not unprecedented for an LCMS method to became the new gold standard.
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