I had a small holding but out now. From the questions from the...

I had a small holding but out now. From the questions from the fda to odac it seems:

1. Problem with sample size
Not all patients were recruited.
Many patients (35%!) did not have the t3151 mutation confirmed at the beginning of the study. This is a patient eligibility criteria so they should not be in there. This makes the sample size issue very difficult for CXS.

3. Primary endpoint
The FDA highlights that they told the company that cytogenetic endpoint was necessary for CML-CP not hematologic.

2. Poor response on primary endpoint
The FDA does not like the resonse rate or duration on the primary endpoint.

3. The indication is patients with the t3151 mutation who failed or did not tolerate imatinib but NO information on the test was provided to the FDA. IT is not a commercial test and in fact the FDA says there were different sites and methods used.

4. Poor management of the study
Some issues were raised eg many patients did not have their t3151 status tested at the beginning of the study and some did not have marrows sent to the central lab.

There is only one pivotal trial so its all or nothing on this one. My suspicion is they will need to do another trial, work out how to handle the t3151 test issue and and follow the prototcol this time. Another capital raise will be required.

http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM199561.pdf