I’m not going to pretend to know what’s at stake here, especially with kids who are cursed with muscular dystrophy. But the FDA advisory panel voted against approval for a drug that was, apparently, being tested without the full benefits of science. Critics argue the trials were too small–because parents didn’t want to sign kids to a trial that potentionally would give them a placebo instead of an active drug.
It’d be the equivalent to signing a death warrant for your child, all for the love of science.
Shares have risen like a Phoenix since the lows set this morning, based upon the belief that the FDA will cave into political pressures and approve the drug, in spite of the board advising otherwise.
Really? Has everyone gone mad?
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Much better, nailed it. It’s a case where the drug most likely works (producing some dystrophin) but the data is a mess (inconsistent biopsies, etc). Add in high-pressure advocacy and less than stellar management, the result is you get a 4-year chart like SRPT’s.
If it looks like it likely works, maybe they could give some kind of conditional approval or something and let the kids have it. As you mentioned, nobody want to sign a death warrant for their child, and you can’t blame anyone for that.
Given their are no other treatements (per my understanding), I agree that conditional approval would be a good idea, unless their are statistically significant, non-reversible side effects.