Clinical Review of Priority Review - Berinert, March 6, 2008

Clinical Review of Priority Review Request for ORIGINNAL BLA

OBRR/DH

BLA  12528                LOGIN ID --b(4)---

 

See also:  IND --b(4)---         

 

Title:  C1-Esterase Inhibitor (Human), Pasteurized

 

Proposed Use (Indication):  For the treatment of acute attack episodes in patients with hereditary angioedema (HAE)

 

Sponsor: CSL Behring LLC

 

Sponsors Point of Contact: Hartmann RPh, Paul R

Address: Director, Regulatory Affairs
                        CSL Behring LLC
                        1020 First Ave 
                        PO Box 61501
                        King of Prussia, PA 19406-0901

 

Phone: 6108784644

Fax:   164108784182

           

CBER Receipt Date: 07 March 2008          

Letter Date:  06 March 2008

Fast Track Application:  No.

Fast Track Request Denied and Denial Communicated to Sponsor

Priority Review Status:  Requested by sponsor with supporting information in Attachment 1, Module 1.

Orphan Drug Status: Granted 16 October 1992 for “Prevention and/or treatment of acute attacks of hereditary angioedema.”

PREA Status:  Full waiver requested in cover letter to BLA, due to orphan drug designation.

Gene Therapy, Somatic Cell Therapy or Xenotransplantation: N/A

Product name(s)/Product Type:

C1-Esterase Inhibitor, Pasteurized

Product Type:  BLOOD/Sub Type: OTHER/C1-INHIBITOR

Review Team:

Felice D’Agnillo (CMC reviewer Committee Chair)

L. ROSS PIERCE  (Primary Medical Reviewer)

CHINYING WANG  (Biostatistics Reviewer)

MARY PADGETT  (CMC  Reviewer)

PAUL BUEHLER  (Product Reviewer)

BHANU KANNAN (BIMO)

Elena Karnaukhova (CMC)

Mahmood IFTEKHAR (Human PK)

NANNETTE CAGUNGUN  (CSO)

PAUL BUEHLER (Pharm/Tox Reviewer)

RECOMMENDATION:

This BLA may be granted FAST TRACK STATUS.

A BPAC should tentatively be convened in August 2008 to help the agency consider the application.

REVIEW

The sponsor correctly states there is no specific therapeutic agent licensed or approved in the U.S. for treatment of acute hereditary angioedema (HAE) attacks.  This meets the usual FDA criteria for priority review.