From: Cagungun, Nannette
Sent: Wednesday, January 12, 2011 6:04 PM
Subject: FXIII Questions from January 11, 2011 telecon
Here are the clinical and statistical questions and comments that were discussed during the telecon yesterday:
- Please adjust the safety data (tables, graphs) to reflect the new total number of subjects, as of the 3-month safety update submission.
- Please confirm the total number of infusions administered. Section 220.127.116.11 states that 3284 doses were administered. Tables 2 (page 26) and 3 (page 27) in Section 2.7.4 of the Clinical Summary confirm the number as 3284, with 72 doses given for Study 3001 and 9 doses given for Study 3002. However, Table 10 in Section 12.1 in 18.104.22.168.2.2 indicates that 81 doses were given for Study 3001 and the data listing (da.xpt) for Study 3002 indicates that 5 doses were given.
Please provide the number of doses administered for the studies in the original submission, the number of additional doses in the 3-month safety update, and the total number of doses.
- Please ensure that the Highlight section of the PI does not exceed the requirement of half a page. Please notify CBER in case you need to expand it so that CBER could apply for a waiver from this requirement.
- Thrombo-embolic and immunogenic events that have occurred during the clinical development are important AEs and therefore must be reflected in the PI in more detail.
- Please provide the completion date for the PMR Phase 4 study; the date of the last subject’s follow-up and the projected date of the Final Study report, as well as the planned number of subjects.
In the interest of time, we would like to receive the responses to these questions by COB on Friday, January 14, 2011. Please send your response as an amendment to the file.
Nannette Cagungun, MS, PD, RAC Regulatory Project Manager OBRR/CBER/FDA, HFM-380 1401 Rockville Pike Rockville, MD 20852-1448 Tel: (301) 827 6174 Fax: (301) 827 2857 Email: email@example.com