Request for Information, November 25, 2013 - Eloctate

From:    Pracht, Leigh
Sent:      Monday, November 25, 2013 4:45 PM
To:         'Debra Segal'
Subject: RE: BLA 125487 - Clarification of FDA Request for Information

Importance:        High

Deb,

No, this approach is not acceptable with FDA.  We believe it will be most useful if Biogen submits the updated PVP prior to both the Late Cycle Meeting and label negotiations.

Best regards,

Leigh A. Pracht 
Regulatory Project Manager
FDA/CBER/OBRR/DBA
WOC1; RM562N; HFM-380
1401 Rockville Pike
Rockville, MD 20852
Telephone: 301-827-6116
BlackBerry:  --b(6)---------
Fax: 301- 827-2857
Leigh.Pracht@fda.hhs.gov

From: Debra Segal [mailto:Debra.Segal@biogenidec.com]  
Sent: Wednesday, November 20, 2013 5:50 PM 
To: Pracht, Leigh 
Subject: RE: BLA 125487 - Clarification of FDA Request for Information

Dear Ms. Pracht, 
Thanks very much for your feedback. 

As Biogen Idec plans to align the text in the PVP with the USPI, we propose to submit the updated PVP following completion of the labeling negotiations.  Is this approach acceptable to the Agency? 

Thanks in advance,
Deb

From: Pracht, Leigh [mailto:Leigh.Pracht@fda.hhs.gov]  
Sent: Wednesday, November 20, 2013 10:52 AM 
To: Debra Segal 
Subject: RE: BLA 125487 - Clarification of FDA Request for Information 
Importance: High

Ms. Segal,

The proposed changes to the Pharmacovigilance Plan (PVP), and the timelines for completion of the ongoing Pediatric (8HA02PED) and Extension (8HA01EXT) studies have been reviewed. At this time FDA does not require additional information. Please submit an updated PVP including the proposed changes.

Best regards,

Leigh A. Pracht 
Regulatory Project Manager
FDA/CBER/OBRR/DBA
WOC1; RM562N; HFM-380
1401 Rockville Pike
Rockville, MD 20852
Telephone: 301-827-6116
BlackBerry:  --b(6)------
Fax: 301- 827-2857
Leigh.Pracht@fda.hhs.gov

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From: Debra Segal [mailto:Debra.Segal@biogenidec.com]  
Sent: Monday, November 18, 2013 2:24 PM 
To: Pracht, Leigh 
Subject: RE: BLA 125487 - Clarification of FDA Request for Information

Hi Leigh,
Thanks for your prompt response regarding the PUP study. 

Are you able to comment on whether our prior responses regarding the PVP are acceptable to you?

Biogen Idec submitted responses to these questions on 09 September 2013 (Sequence No. 0017).  The responses included proposed changes to the Pharmacovigilance Plan (PVP), as well as projected timelines for completion of the ongoing Pediatric (8HA02PED) and Extension (8HA01EXT) studies.  Has the Agency had a chance to review these 09 September responses, as 
we believe that they address the Agency’s feedback?   
Thanks in advance, 
Deb

From: Pracht, Leigh [mailto:Leigh.Pracht@fda.hhs.gov]  
Sent: Monday, November 18, 2013 1:58 PM 
To: Debra Segal 
Subject: RE: BLA 125487 - Clarification of FDA Request for Information 
Importance: High

Deb,

Your proposal to conduct a separate trial in previously untreated patients (PUPs) is acceptable.  We will consider this PUPs trial as a Postmarketing Commitment Study.  Please submit timelines for the submission of the study protocol, completion of study, and submission of interim and/or final study reports.

Best regards,

Leigh A. Pracht 
Regulatory Project Manager
FDA/CBER/OBRR/DBA
WOC1; RM562N; HFM-380
1401 Rockville Pike
Rockville, MD 20852
Telephone: 301-827-6116
BlackBerry:  --b(6)---------
Fax: 301- 827-2857
Leigh.Pracht@fda.hhs.gov

From: Debra Segal [mailto:Debra.Segal@biogenidec.com]  
Sent: Sunday, November 17, 2013 11:07 PM 
To: Pracht, Leigh 
Subject: RE: BLA 125487 - Clarification of FDA Request for Information

Dear Leigh, 
I am writing to follow up with you regarding our request for clarification regarding the 11 September 2013 FDA Request for Information (BLA 125487).  A copy of the email request is provided below for your 
convenience.As our responses are due to the Agency on 1 December, it would be very helpful to receive your feedback by Wednesday, 20 November. 

Kind regards, 
Deb

Debra Segal
Director, Regulatory Affairs
Biogen Idec Hemophilia
781-464-1858 (work)
--b(6)--------------

From: Debra Segal  
Sent: Sunday, October 20, 2013 9:42 PM 
To: Leigh A. Pracht (Leigh.Pracht@fda.hhs.gov) 
Subject: BLA 125487 - Clarification of FDA Request for Information

Dear Leigh, 
I am writing to you regarding the Agency’s Request for Information, dated 11 September 2013, which was sent to Biogen Idec following our mid-cycle review teleconference.  Specifically, I am seeking clarification regarding questions 31 and 32 in the Clinical/Pharmacovigilance section.  These questions are very similar to those from the earlier Request for Information, dated 26 August 2013.  

Biogen Idec submitted responses to these questions on 09 September 2013 (Sequence No. 0017).  The responses included proposed changes to the Pharmacovigilance Plan (PVP), as well as projected timelines for completion of the ongoing Pediatric (8HA02PED) and Extension (8HA01EXT) studies.  Has the Agency had a chance to review these 09 September responses, as we believe that they address the Agency’s feedback?  

I am also seeking clarification regarding the Agency’s request (Question #31; 11 September) that we expand the inclusion criteria for the ongoing pediatric study to include previously untreated patients (PUPs).  As noted in our 09 September Response to Request for Information (Table 2), a future study of rFVIIIFc in PUPs is planned.   However, in our recent late-cycle meeting for BLA 125444 held on 12 September, the Agency stated that a study in PUPs was not a post-marketing commitment (PMC).  Is this also the case for rFVIIIFc?  

Thanks in advance for your assistance with these matters. 

Kind regards, 
Deb
