Indeed, the co primary endpoint of the TKT/Shire's Hunter trial (and also of the GENZ's MPS-I extension trial) was pulmonary function as measured by FVC and functional capacity as measured by 6MW test.
On Pompe - the infantile onset form is the most severe one, these babies hardly make it to the end of their first year. Since Myozyme has demonstrated survival advantage (even though it isn't a cure) in this setting, drug from the 160L scale is prescribed for infants and they all get the therapy.
The adult form (about 80% of patients), is late-onset, can be undetected for many years, sometimes misdiagnosed and severity &life span are variable. The general clinical progression of untreated late-onset Pompe disease is not well defined. Clinical response to ERT among late-onset patients varies much and endpoints are not optimal for tracking disease progression. So docs have to evaluate severity, benefits, risk and cost on a case-by-case basis.
As for the LOTS trial: (btw, I wrote you my take on the 12/14/2007, in a PM) If data had shown no signals of efficacy at all or a new safety issue, it would have worried me but given the above, I think that even suggestions of activity is good enough. Docs are currently treating some of those adult patients off-label with Myozyme and they probably will not stop.
Panel could still recommend for approval and anyway, like we read through the briefings, GENZ will have few options to use Myozyme from the 2000L facility and charge for it.
PS Thanks for your reply on the ITMN vs Shionogi trials :)
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