Replies to post #141202 on Biotech Values

[DewDiligence]

TKT had a tough sell with the FDA following the disparate advisory-panel votes for Fabrazyme and Replagal in 2003:

http://www.thepharmaletter.com/file/17117/fda-panel-backs-fabrazyme-but-not-replagal-for-fabry-disease.html

[jq1234]

I know quite well. I might be biased because I had direct interest with one party at that time. I strongly believe FDA would have granted approval to both similar to EU if the trial designs were similar and benefits were similar. FDA advisory panel voted 15-0 Replagal trial had NOT provided substantial evidence that Replagal was effective - the vote on Fabrazyme was 14-1 in favor. To me, it wasn't orphan drug status of Fabrazyme that kept Replagal off US market. It's in their clinical data. Even this year, they had to withdraw Replagal BLA.

EMA granted Fabrazyme approval under ‘Exceptional Circumstances’ which ended only in 2008 - convert to full approval.

[genisi]

In EU, Fabrazyme and Replagal applications were submitted on the same week for EMEA approval and were approved on the same day with co-exclusivity marketing authorisations. In the US, applications were submitted on the same month but the FDA approved Fabrazyme first and with approval came orphan drug marketing exclusivity that TKT since had to break.