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Vaccines, Blood & Biologics

Filing Meeting Summary - Berinert, April 18, 2008

Filing Meeting Summary


Date:               April 18, 2008                       Time:  3:00 PM


From:              Nannette Cagungun, CBER/OBRR/DBA, HFM-380


To:                  STN 125287/0


Subject:          Filing Meeting for CSL Behring’s C1 Esterase Inhibitor BLA


CBER Participants:

Nannette Cagungun

Paul Buehler

Omer Butt

Felice D’Agnillo

Dave Doleski

Mahmood Farshid

Basil Golding

Elena Karnaukhova

Ross Pierce - Absent

Jean Mackie

Bhanu Kannan

Jean Wang      



The sponsor submitted the BLA on March 7, 2008.  They are requesting a priority review of the BLA.  The product has been on the market overseas for more than twenty years, where it is known as Berinert.   



The filing meeting was held today with everyone in attendance except for the clinical reviewer. 



There are two statistical and clinical issues that have seriously impeded the review of the BLA and have delayed the early submission of a BIMO inspectional assignment.


1.  CSL Behring (CSLB) has not yet provided a list of the definitions of the field variable names for the clinical safety and efficacy and PK databases. 


2. CSLB did not provide an analysis-ready dataset for each summary table presented for Efficacy and Safety Findings (i.e. Tables 5, 6, 7, 8 in the section of Clinical Overview).  CBER will send an Information Request for the datasets.



CSLB’s response to CBER’s April 15, 2008 Information Request will be reviewed upon its receipt and a meeting will be held soon after to discuss the adequacy of the response.



BIMO has sent an inspection assignment request to the field office.  Inspection of the sites is anticipated in mid-July.  The sites are all located in the U.S.  The inspection report will not be available until late July.



The CMC reviewers recommend the filing of the BLA.

CSLB does not appear to have submitted data on the C4 assay or any validation data for the C1 antigenic assay. CMC reviewers will evaluate the anti-C1INH antibody assay data when those are submitted.

Validation of the functional C1 inhibitor assay for clinical samples has not yet been identified in the submission at this point in time.

The Scientific Lead will prepare the Lot Release Testing Plan.



The Division of Epidemiology reviewer will determine if a pharmacovigilance plan is necessary. 



The pre-approval inspection is scheduled for May 2008.  The CMC product inspector will need to be determined soon.  ORA will not be joining the inspection team.

DMPQ review will be mostly done by mid-cycle.



The BLA can be filed from APLB’s perspective.  CSLB should submit a proprietary name review (PNR) request by mid-cycle.  The applicant should follow the guidance and SOPs if they are considering a tradename.


Action Item:

1.  This BLA will be filed.

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