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Clinical Review of Priority Review - Berinert, March 6, 2008
Clinical Review of Priority Review Request for ORIGINNAL BLA
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
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
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)
This BLA may be granted FAST TRACK STATUS.
A BPAC should tentatively be convened in August 2008 to help the agency consider the application.
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.