Record of Telephone Conversation, March 12, 2013 - ALPROLIX

Submission Type: BLA    Submission ID: 125444/0    Office: OBRR
 Product:          Coagulation Factor IX (Recombinant), Fc Fusion Protein
 Applicant:       Biogen Idec Inc.
 Telecon Date/Time: 12-Mar-2013 01:30 PM        Initiated by FDA? No
 Communication Category:                 1. Advice
 Drafted:                      Edward Thompson
 Reviewed/ Revised:    Nisha Jain, Nancy Kirschbaum, Stephanie Omokaro

Telecon Topic:           Discuss FDA decision for not granting Priority Review, as requested by Biogen Idec
 FDA Participants:       
 Nisha Jain, MD, Chief, CRB/DH/OBRR/CBER/FDA
 Nancy Kirschbaum, PhD, LH/DH/OBRR/CBER/FDA
 Stephanie Omokaro, MD, CRB/DH/OBRR/CBER/FDA
 Edward Thompson, RPMB/DBA/OBRR/CBER/FDA

Biogen Idec Participants: 
 Glenn Pierce, MD, PhD, Senior Vice President, Hemophilia Development
 Aoife Brennan, MB, BCh, BAO, MMSC, Director, Medical Research
 Nadine Cohen, PhD, Senior Vice President, Regulatory Affairs
 Debra Segal, Director, Regulatory Affairs
 Daniel Soroko, MS, Associate Director, Regulatory Affairs
 Paula Cobb, Vice President, Program Leadership and Management

Telecon Body:
 Biogen Idec requested the teleconference to discuss the decision by FDA for a standard review of original biologics license application STN 125444/0.  Biogen Idec had requested Priority Review. 
 In providing a response, FDA referred to available guidance in determining regulatory requirements germane to the designation of Priority Review 1.  Guidance specifically states, “Products regulated by CBER are eligible for priority review if they provide a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease.  FDA communicated that there was no clinical evidence thus far, which indicated that rFIXFc had demonstrated better clinical safety or efficacy than BeneFIX, Mononine or Alphanine commercial products.  The potential benefit from reduced infusion frequency for prophylaxis, while potentially a Quality of Life issue, does not directly impact its safety and efficacy profile.  Furthermore, the ultimate dosing regimens described in the product label will follow critical review of submitted clinical data; therefore, a potential improvement in patient compliance as a result of fewer infusions can only be determined after complete review of the BLA.  In conclusion, although, rFIXFc is unique compared to the other factor IX products, treatment modes are the same and similar safety and efficacy can be expected after each administration; therefore, rFIXFc does not meet an unmet medical need as there is available therapy for the patients.
 END

Footnote:

1 Guidance for Industry: Available Therapy, 2004
