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Biotech / Medical : Biotech Valuation -- Ignore unavailable to you. Want to Upgrade?

To: IRWIN JAMES FRANKEL who wrote (7693)	1/12/2003 4:52:09 PM
From: Biomaven	Read Replies (1) \| Respond to of 52153

IJ, Here's what Reuters says: On Monday, Genzyme will present data on its drug, called Fabrazyme, to a panel of independent FDA advisers. Genzyme said the FDA is not expected to ask the panel to vote on whether to recommend the drug's approval, but will seek other input. The agenda says: 1:30 Committee Discussion and Vote My guess is that the FDA might ask the committee some key questions, like whether they hit their surrogate endpoints (yes, based on my quick review of the documents) and whether the surrogate endpoints mean anything (unknown, but seems plausible to me). I've seen panels before where the vote was not on approval, but instead on some more scientifically oriented questions. I think the FDA will approve based on what I saw. Mostly, it seems the FDA are now whining about whether the surrogate is valid, but seeing as this is the endpoint they agreed to, it's pretty hard for them to grumble about it now. With such a limited diseasae population and a variable, chronic disease, it's always going to be extremely hard, if not impossible, to prove anything based on something other than a surrogate endpoint. I figure the FDA will whine some more and then approve, subject to a Phase IV. Here's the standard, by the way: § 601.41 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity. FDA may grant marketing approval for a biological product on the basis of adequate and well-controlled clinical trials establishing that the biological product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predict clinical benefit or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity. Approval under this section will be subject to the requirement that the applicant study the biological product further, to verify and describe its clinical benefit, where there is uncertainty as to the relation of the surrogate endpoint to clinical benefit, or of the observed clinical benefit to ultimate outcome. Postmarketing studies would usually be studies already underway. When required to be conducted, such studies must also be adequate and wellcontrolled. The applicant shall carry out any such studies with due diligence. Peter