First Committee Meeting Summary - Berinert, March 25, 2008

First Committee Meeting Summary

Date:     March 25, 2008                                 Time:  2:00 PM – 3:00 PM

                                   

From:    Nannette Cagungun

 

To:         STN 125287/0

 

Re:      First Committee Meeting for CSL Behring’s C1 Esterase Inhibitor BLA


 

CBER Participants:

Nannette Cagungun

Paul Buehler- Absent

Omer Butt

Felice D’Agnillo

Dave Doleski

Mahmood Farshid – Absent

Basil Golding

Elena Karnaukhova - Absent

Ross Pierce

Jean Mackie - Absent

Bhanu Kannan – Absent

Jean Wang      

 

Background:

The applicant, CSL Behring GmbH, submitted the BLA for their C1 Esterase Inhibitor on March 7, 2008.  They have requested a priority review of the BLA.  The product has been on the overseas market for at least twenty years, where it is known as Berinert.  The applicant has indicated that they will request a proprietary name review some time in the future for a name that has not yet been determined.

 

Discussion:

Following introductions, Felice D’Agnillo stated that the submission appeared complete from the CMC perspective. He also briefly described the specific review assignments for the CMC review team. The CMC review team noted that there was some inconsistencies in which lots were being designated as conformance lots. The team indicated that this issue would have to be clarified by the sponsor. Felice D’Agnillo also stated on behalf of Paul Buehler, the Pharm/Tox reviewer, that the application seemed complete from a preclinical perspective. Ross Pierce stated that the submission seemed complete from a clinical reviewer’s perspective.  He suggested that CBER ask CSL Behring whether the data submitted to the IND are the same as the data submitted to the BLA. 

 

Dave Doleski indicated that the pre-approval inspection would take place in mid-May.  Marion Michaelis from DMPQ will participate.  The review committee agreed to convey preliminary review issues to the company prior to the filing date.  Concerns regarding the PK study could be conveyed to the applicant now.

 

Ross Pierce discussed the results of robustness analysis of the primary endpoint (time of attack to time of initial relief of symptoms) that OBRR requested in the 20 December 2007 IND letter. He informed the committee that OBRR requested raw data in 2007 so that BIMO review could begin right away.  Inspection sites have already been selected.  The inspection dates are just waiting to be finalized and could take place by the middle of June 2008. It was noted that the results of the BIMO inspections would therefore not be known prior to the date of the Midcycle meeting for this file.

 

Two issues have been identified at this time:
1.The use of PK research data from a doctoral dissertation.  CBER will ask CSLB if FDA could inspect the laboratory where the assays were done.
2.Possibility of presenting the BLA to BPAC in September.  Since the action due date is on September 5, 2008, Dr. Golding indicated he would clarify with upper management whether the BPAC presentation could be waived.

The meeting adjourned.
