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Re: mcbio post# 160398

Saturday, 04/27/2013 4:40:55 PM

Saturday, April 27, 2013 4:40:55 PM

Post# of 257262
VPHM was talking about prophylaxis of HAE because they were in discussion with FDA on the development of subQ version of Cinryze. They mentioned large controlled trial, large safety database, maybe 100 if I remember correctly, which is VERY large for tiny orphan indication like this, plus just for a different version of their own drug. Cinryze was approved based on 20 something patients originally.

As of advantage, I think you misunderstood genisi's post. She wasn't talking about DX2930 specifically, rather what would be needed for a successful drug for prophylaxis of HAE, which is a very high bar. You can't predict those based on preclinical data, especially for a drug with same mechanism but in different form of Kalbitor that has a black box warning. Genzyme terminated partnership with Dyax on Kalbitor in 2007. It was a good decision.

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