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Vaccines, Blood & Biologics

Mid-cycle Meeting Summary, November 16, 2010 - Corifact

Date:               November 16, 2010                         Time:  1:00 PM – 2:00 PM

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

To:               STN 125385/0

Subject:        Mid-cycle Meeting for CSL Behring’s Factor XIII BLA

CBER Participants:
Nannette Cagungun
Basil Golding
Nisha Jain
Timothy Lee
Daniela Vanco
Ze Peng
Iftekhar Mahmood
Alan Ou
La’Nissa Brown
Dennis Cato
Renee Rees
Michael Brony – by phone
Stephanie  Omokaro
Jennifer Schmidt – absent
Martha O’Lone – on leave


The scientific lead, Dr. Ze Peng, gave an update on the status of this BLA.  The BLA was filed on October 12, 2010.  No major CMC issues have been identified but an information request will most likely be sent to the applicant before the end of the month.  The lot release protocol is now under review in the Product Release Branch (PRB).  Dr.  Peng is currently working with the Division of Product Quality (DPQ) on the lot release testing plan.  There will be no PMR for the -------------------(b)(4)-------------------.  As agreed upon during the pre-BLA meeting, CSL Behring will ------------------------------(b)(4)--------------------------------------------------- after the approval of the BLA.  Because this product has an orphan designation, the BLA will not be presented to the PERC.  Dr. Peng will evaluate the need for an information request for the final validation report on the ------------(b)(4)--------------.

Dr. La’Nissa Brown indicated that she did not find any preclinical issues with the BLA.  She will provide comments for the package insert.

Dr. Iftekhar Mahmood noted that one PK parameter appears to be incorrect.  He will send his comments to the RPM and the Scientific Lead so these could be conveyed to CSL Behring.  He has provided labeling comments for the package insert in his review memo. 

Dr. Daniela Vanco remarked that the product appears to have a favorable safety profile.  She will look more closely into the serious adverse events reported in the submission particularly as they relate to thromboembolic events.  

Dr. Alan Ou stated that CSL Behring’s pharmacovigilance plan (PVP) is acceptable.  He recommended that CSL Behring continue with their routine pharmacovigilance activities with additional activities they themselves have proposed for certain spontaneously reported cases, such as: 1) collecting more detailed information about certain spontaneously reported cases with identified risks, 2) following up the cases with a targed questionnaire, 3) collecting general information about the patient to characterize AEs and identified risks.  In addition, CSL Behring should consider expanding the safety database to include certain groups, such as pregnant and lactating women, children younger than 2 years, and and the geriatric population.

Dr. Renee Rees indicated that an analysis of bleeding events was not presented in the submission because the pivotal study in support of the BLA has a PK surrogate efficacy endpoint.   She has discussed with the clinical reviewer the need for OBRR criteria to use for determining the post-marketing study’s success and failure regarding bleeding events. She will next focus her efforts on reviewing the safety data (for both treatment and FFP groups) submitted in the IND. 

---------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------(Information withheld per the Privacy Act)---------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------.

There are no labeling issues to date.  CBER notified CSL Behring last week that the proprietary name, Corifact, is acceptable.  Dr. Michael Brony is now reviewing the product’s packaging from an advertising and promotional perspective.

A discussion of the timeline for the summary basis for regulatory action (SBRA) ensued.  The members of the review team will provide the scientific lead with an executive summary of their review by the end of December.  Dr. Peng expects to finish the first draft by January 7, 2011; the second draft by January 14, 2011; and the third draft which will be sent to Dr. Golding by January 21, 2011.  The draft will then be sent to OBRR’s deputy director for concurrence by the first week of February 2011.

The meeting adjourned.


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