Major Amendment Memorandum - ALPROLIX

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Public Health Service
 Food and Drug Administration
 Center for Biologics Evaluation and Research


 From:  Nancy Kirschbaum, PhD, Committee Chair and Product Discipline Reviewer
 Ellen Huang, Facilities Discipline Reviewer

To:  Basil Golding, MD, Director, Division of Hematology, OBRR

Subject:  Designation of BL STN 125444/0.45 as a Major Amendment

Designation of Amendment 125444/0.45 as Major
BL STN 125444/0 for Coagulation Factor IX (Recombinant), Fc Fusion Protein was submitted by Biogen Idec, Inc. (Biogen) on 28 December 2012 under the PDUFA V program. The action due date for the BLA is 27 December 2013. On 20 November 2013, Biogen submitted amendment 125444/0.45 containing lyophilization validation study protocols and reports, which were the culmination of an ongoing dialogue between Biogen and the Agency regarding studies and documentation required for complete lyophilization validation. On behalf of the review committee for BL STN 125444/0, we recommend designation of 125444/0.45 as a major amendment.

Justification for Major Amendment Designation
SOPP 8402 defines a major amendment as a submission of information to a pending application that extends the review clock. According to section V.D.2., a major amendment may contain a substantial amount of new manufacturing or facility information not previously submitted to or reviewed by the Agency. Throughout review of BL STN 125444/0, the Agency and Biogen have had ongoing dialogue concerning lyophilization validation studies that needed to be performed and the submission of reports from those studies to the BLA. On 20 November 2013, Biogen submitted study protocols and accompanying final reports as amendment 125444/0.45. Amendment 125444/0.45 was received five (5) weeks before the action due date and represents substantive information and data pertaining to lyophilization validation. As such, amendment 125444/0.45 may be classified according to CBER policy stated in section V.H. of SOPP 8402, as containing substantive information that was received so near the action due date as to preclude adequate time for complete review.

New Action Due Date
According to section V.E.1.b. of SOPP 8402, a major amendment extends the action due date of an original application by three (3) months. If the major amendment designation receives concurrence by the Director, Division of Hematology, OBRR, the new action due date will be 27 March 2014.


 Footnote:

1SOPP 8402: Designation of Amendments as Major, Version #4, October 1, 2012
