Record of Telephone Conversation, May 13, 2014 - Eloctate

Submission Type: BLA    Submission ID: 125487/0    Office: OBRR
Product:          Antihemophilic Factor (Recombinant), Fc Fusion protein
Applicant:       Biogen Idec Inc.

 Telecon Date/Time: 13-May-2014 01:00 PM        Initiated by FDA? No
Communication Category:     1. Advice

Drafted: Edward Thompson
Revised:  Lisa Faulcon

Telecon Summary:     Discuss insufficient data to support package insert label for dose regimen of weekly prophylaxis.

FDA Participants:
Nisha Jain, MD, Chief, Clinical Review Branch, OBRR/DH
Lisa Faulcon, MD, OBRR/DH/CRB
Nancy Kirschbaum, PhD, OBRR/DH/LH
Edward Thompson, OBRR

Non-FDA Participants:
Biogen Idec Inc.
Aoife Brennan, Senior Director, Clinical Development
Ivan Nestorov, Senior Director, Pharmacometrics
Lynda Cristiano, Senior Director, Drug Safety
Yingwen Dong, Lead Biostatistician
Suzanne Stella, Director, Regulatory CMC
Marion Scocca, Director,  Regulatory Affairs
Debra Segal, Director, Regulatory Affairs

Telecon Body:
Biogen Idec Inc. requested this teleconference to discuss the information request sent on May 8, 2014  They specifically requested a better understanding of the requested change to delete the Weekly Prophylaxis Regimen language in the package insert.

FDA stated that the data in the application did not support the labeled dose regimen of weekly prophylaxis because the results of the individualized prophylaxis arm showed that subjects treated with ELOCTATE require a median dose of 50 IU/kg given every 4 days. In addition, the annualized bleeding rate, as well as the number of subjects that were discontinued due to adverse events, and that had bleeding rates greater than 20 bleeds per year were higher for subjects treated with weekly prophylaxis, as compared to subjects treated with individualized prophylaxis. FDA advised that a weekly prophylaxis indication would need to be evaluated in an additional clinical study.  FDA stated that the efficacy results for weekly prophylaxis will be included in the clinical trials section label.
END