Vaccines, Blood & Biologics
Our STN: BL 125287/110
CSL Behring GmbH
Attention: Mr. Paul R. Hartmann
CSL Behring LLC
1020 First Avenue, P.O. Box 61501
King of Prussia, PA 19406-0901
Dear Mr. Hartmann:
We have approved your request to supplement your biologics license application for C1 Esterase Inhibitor (Human), to include a revision of the indication for the product to read, “…for the treatment of acute abdominal, facial, or laryngeal attacks of Hereditary Angioedema (HAE) in adult and adolescent patients”;and the submission of the PMR Final Study Report under 505(o) - Completion of Planned Ongoing Open Label Extension Study CE1145_3003 (IMPACT II) for laryngeal HAE attacks.
The review of this product was associated with the following National Clinical Trial (NCT) number: NCT00292981.
Under 21 CFR 201.57(c)(18), patient labeling must be reprinted at the end of the package insert. We request that the text of information distributed to patients be printed in a minimum of 10-point font.
Please provide your final content of labeling in Structured Product Labeling (SPL) format and include the carton and container labels. In addition, please submit three original paper copies for carton and container final printed labeling. All final labeling should be submitted as Product Correspondence to this BLA at the time of use (prior to marketing) and include implementation information on FDA Form 356h [OPTION: and FDA Form 2567 as appropriate].
In addition, please submit the final content of labeling (21 CFR 601.14) in SPL format via the FDA automated drug registration and listing system, (eLIST), as described at http://www.fda.gov/ForIndustry/DataStandards/StructuredProductLabeling/default.htm. Information on submitting SPL files using eLIST may be found in the guidance for industry titled, “SPL Standard for Content of Labeling Technical Qs and As at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM072392.pdf.
You may submit two draft copies of the proposed introductory advertising and promotional labeling with an FDA Form 2253 to the Center for Biologics Evaluation and Research, Advertising and Promotional Labeling Branch, HFM-602, 1401 Rockville Pike, Rockville, MD 20852-1448. You must submit copies of your final advertisement and promotional labeling at the time of initial dissemination or publication, accompanied by Form FDA 2253 [21 CFR 601.12(f)(4)].
All promotional claims must be consistent with and not contrary to approved labeling. You should not make a comparative promotional claim or claim of superiority over other products unless you have substantial evidence or substantial clinical experience to support such claims [21 CFR 202.1(e)(6)].
Please submit an amendment to all pending supplemental applications for this BLA that include revised labeling incorporating a revised content of labeling that includes these changes.
This fulfills your commitment to submit the final study report under 505(o) - Completion of Planned Ongoing Open Label Extension Study CE1145_3003 (IMPACT II) for laryngeal HAE attacks as stated in commitment number 2 of the October 9, 2009 approval letter.
Under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), all applications for new active ingredients, new indications, new dosage forms, new dosing regimens, or new routes of administration are required to contain an assessment of the safety and effectiveness of the product for the claimed indication in pediatric patients unless this requirement is waived, deferred, or inapplicable.
Because the biological product for this indication has an orphan drug designation, you are exempt from this requirement.
We will include information contained in the above-referenced supplement in your biologics license application file.
Basil Golding, M.D.
Division of Hematology
Office of Blood Research and Review
Center for Biologics
Evaluation and Research