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Memorandum of Teleconference, August 20, 2009 - Hizentra

Memorandum of Teleconference dated August 20, 2009 between CSLB and FDA

Date of Telecon: 8/20/09 Time: 09:30 - 09:55

Purpose of Telecon: to discuss Pediatric Assessment submission for BLA 125350/0

FDA Participants: Hon-Sum Ko and Nisha Jain

CSLB Participants:
Paula Hines, PhD, Regulatory Affairs, King of Prussia, PA
Dominique Schaller, PhD, Regulatory Affairs, Bern, Switzerland
Mikhail Rojavin, PhD (Clinical R&D), King of Prussia
Peter Kiessling, PhD (Clinical R&D), Marburg, Germany.

Background:
For BLA STN 125350, the pediatric subjects studied include 3 children and 7 adolescents. CSLB has an ongoing study in Europe that includes 18 children and 5 adolescents. CSLB has been asked to request deferral of submission of pediatric assessment for the children and adolescent age groups in the treatment of primary immunodeficiency. The firm would like to submit the European data prior to approval.

Discussion:
FDA reiterated that the best course of action is to ask for deferral of submission of the European pediatric data.

CSLB asked what kind of labeling can be expected if deferral is requested. An example such as that for Flebogamma was discussed, and this would be the most likely outcome.

FDA explained to CSLB that all information relating to pediatric data will be reviewed by the Pediatric Review Committee, which requires advance scheduling and review of the application up to 3 months prior to approval. Submission of European data prior to approval would make the time frames very difficult to accommodate consideration for this application by the Committee.

CSLB asked about how to integrate the European data with the U.S. data already in the application. FDA advised the firm to refer to the Integrated Summary of Effectiveness Guidance (Aug 2008) and the document Placement of Integrated Summaries of Safety and Effectiveness (ISS/ISE) in Applications Submitted in the eCTD Format. Safety data, especially, should be submitted in totality for our evaluation.

The call ended amiably.