Re: Myozyme and FoB’s
>I take this to mean that GTCB would need to perform complete trials and NDA for their FOBs…<
I assume by complete trials you mean a full-fledged clinical-development program as in an NDA or BLA. If so, then I do not agree with your supposition.
The FDA is not making GENZ run a full-fledged clinical program for the new version of Myozyme; to the contrary, the FDA is merely asking that GENZ supplement its dossier for the old version of Myozyme with data on safety/immunogenicity of the new version.
This is IMO exactly the way investors should expect the FDA to behave for typical FoB applications once a regulatory path is established by Congress.
>(i.e they could not use the generic ANDA mechanism).<
The ANDA mechanism applies only to drugs regulated as small molecules (although by historical accident it includes carbohydrate drugs and certain small-protein drugs). FoB’s will eventually be regulated under a separate statutory provision that will presumably have some of the same features as ANDA’s and 505b2 NDA’s.
>From what I understand even though Atryn is different in terms of its carbohydrate pattern from the native protein the bioactivity is the same and the it has not stimulated an immune response in any patient to date.<
That’s correct, but it’s important to bear in mind that the ATryn is not an FoB. Rather, GTC has run a full-fledged BLA program demonstrating non-inferiority of Atryn to the approved plasma-derived antithrombin. Antibody data will be included in the ATryn BLA, but the FDA decision will be based on ATryn’s own dossier rather than the dossier of a comparator drug. Regards, Dew
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