More evidence of a shift in the winds at FDA, here. . . .

[UPDATED 04.2830.08 AM -- Another very-well researched, and well-written piece (from that shiny-new pen of PM, at Gooznews) on the changing tides at FDA. . . .

4.28.08: As will, no doubt, be the case, repeatedly, in the future, Pharmalot is on this, now -- and he's done a bang-up job of making it easy to read, and yet coveying all the essence of it. . . Kudos!

And, from the very same friend of this blog as below -- this would seem to confirm all of the below.]

This just in, from a friend of this blog (Thanks!) -- and, as additional evidence of an emerging trend toward more rigorous review-before-approval at FDA -- I've been alerted to the fact that FDA told Isis and Genzyme that they will need to conduct a study with clinical endpoints before FDA will even consider approval¹. The drug is Mipomersen (formerly Isis 301012). This is plainly significant, because the companies clearly had been hoping to get approval based on LDL-lowering alone. I think this reflects new thinking at FDA on clinical v. surrogate endpoints -- and that, in turn, sounds a lot like FDA has learned some "hard lessons" from the ENHANCE study train-wreck.

From the Genzyme Form 8-K, of April 23, 2008:

. . . .Mipomersen for high-risk cardiovascular disease

Genzyme expects to finalize its license of mipomersen from Isis Pharmaceuticals in the second quarter and subsequently communicate a development plan for the product. Mipomersen is a lipid-lowering agent being developed primarily for patients at significant cardiovascular risk who are unable to achieve target cholesterol levels with statins alone or who are intolerant of statins. The product offers an innovative approach to addressing a serious unmet medical need, and Genzyme believes it could prove to be the most effective lipid-lowering agent for high risk cardiovascular disease patients for whom conventional therapies are not sufficient. The product may provide significant benefit over the standard of care and targets a well-defined and severely ill patient population. Mipomersen is currently being studied in a Phase 3 clinical trial involving patients with homozygous familial hypercholesterolemia. . . .

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[Footnote 1: This FDA directive does not affect the accelerated approval track of the drug, for homozygous familial hypercholesterolemia patients.]