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Vaccines, Blood & Biologics

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Fast Track Development Program, August 27, 2010 - Corifact

Designation and Review Programs
SOPP 8414
Appendix 7
Review for Determining Fast Track Development Program Designation
________________________________________________________________

Review for Determining Fast Track Development Program Designation

Receipt Date of Fast Track Request: 08/18/2010 (?)       Date of Review Memo: August 27, 2010

BLA number: 125385                                                          Amendment number: 0

Product: Factor XIII Concentrate (Human)

Sponsor: CSL Behring

Condition for which the drug is intended and the specific anticipated benefits of use: The proposed indication for Factor XIII Concentrate is routine prophylactic treatment of congenital Factor XIII deficiency.

A.  Consideration of Fast Track Elements:                                                                     

 

  1. Is the aspect(s) of the condition anticipated to be benefited serious or life-threatening?                 X Yes         No    
  2. Does the drug show potential (given its stage of development) to treat this serious aspect of the condition?       X Yes         No 
  3. Is the drug development program designed to determine whether the drug will   affect a serious aspect of  the condition?  (Degree of specificity should be appropriate to the stage of development)    X Yes   No 
  4. Is there any accepted/approved treatment for the same serious or life-threatening   aspect of the condition          being studied? Yes         X No

Recommendation: The Division of Hematology is granting the fast track designation under 21 CFR 601, Subpart E, §601.41 for the submitted clinical development.

The Factor XIII Concentrate is intended to treat a serious and life-threatening condition; Factor XIII deficiency is a rare hereditary bleeding disorder of high life-threatening potential. Although cryoprecipitate and fresh frozen plasma provide a source of factor XIII, these products carry risks of blood-born disease, viral transmission, fluid overload, immunologic reactions, etc. There are only two commercial Factor XIII products in Europe, which are not approved in the US.  Fibrogammin-P (CSL Behring UK Limited) is available in the United States only under IND/clinical ---------------(b)(4)--------------------------------, or for prophylactic intervention. There is currently no approved factor XIII replacement therapy in the US. Therefore the product is addressing an unmet medical need.

FDA may grant marketing approval for this biological product based on the submitted adequate and well-controlled trial establishing the effect of Human Plasma-derived Factor XIII concentrate on a surrogate endpoint (Factor XIII through activity levels).

The Sponsor was notified that the approval under this section will be also subject to the post-marketing requirement safety and efficacy study to prove the relation of the surrogate endpoint to clinical benefit. This study is ongoing (under BB-IND (b)(4)).

      Fast Track Development Program is (check one)

      granted_____X______

      denied_____________

     
Reviewer Signature/Date: Daniela J. Vanco, M.D./August 27, 2010
Concurrence by Branch Chief:

Signature/Date________________________________________

     
Concurrence by Division Director:

Signature/Date________________________________________