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December 4, 2009 Approval Letter - Wilate
Our STN: BL 125251/0
Octapharma Pharmazeutika Produktionsges.m.b.H.
Attention: Mr. Stanley Ammons
Octapharma USA, Inc.
121 River Street, 12th floor
Hoboken, NJ 07030
Dear Mr. Ammons:
We have approved your biologics license application for von Willebrand Factor/Coagulation Factor VIII Complex (Human) effective this date. You are hereby authorized to introduce or deliver for introduction into interstate commerce von Willebrand Factor/Coagulation Factor VIII Complex (Human) under your existing Department of Health and Human Services U.S. License No. 1646. Your product, von Willebrand Factor/Coagulation Factor VIII Complex (Human), is indicated for the treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated.
Under this authorization, you are approved to manufacture von Willebrand Factor/Coagulation Factor VIII Complex (Human) at your facility in Vienna, Austria. You may label your product with the proprietary name Wilate® and market it in 450 and 900 IU/vial fill sizes.
We did not refer your application to the Blood Products Advisory Committee because our review of information submitted in your BLA, including the clinical study design and trial results, did not raise concerns or controversial issues that would have benefited from an advisory committee discussion.
The dating period for von Willebrand Factor/Coagulation Factor VIII Complex (Human) shall be 36 months from the date of manufacture when stored at 2 to 8 °C. The date of manufacture shall be defined as the date of final sterile filtration of the formulated drug product. Following the final sterile filtration, no reprocessing/reworking is allowed without prior approval from the Agency.
Please submit final container samples of the product in final containers together with protocols showing results of all applicable tests. You may not distribute any lots of product until you receive a notification of release from the Director, Center for Biologics Evaluation and Research (CBER).
You must submit information to your biologics license application for our review and written approval under 21 CFR 601.12 for any changes in, including but not limited to, the manufacturing, testing, packaging or labeling of von Willebrand Factor/Coagulation Factor VIII Complex (Human), or in the manufacturing facilities.
You must submit reports of biological product deviations under 21 CFR 600.14. You promptly should identify and investigate all manufacturing deviations, including those associated with processing, testing, packing, labeling, storage, holding and distribution. If the deviation involves a distributed product, may affect the safety, purity, or potency of the product, and meets the other criteria in the regulation, you must submit a report on Form FDA-3486 to the Director, Office of Compliance and Biologics Quality, Center for Biologics Evaluation and Research, HFM-600, 1401 Rockville Pike, Rockville, MD 20852-1448.
Please submit all final printed labeling at the time of use and include implementation information on FDA Form 356h and FDA Form 2567 as appropriate. Please provide content of labeling in Structured Product Labeling format. Please provide three original paper copies for all other labels.
In addition, you may wish to 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.
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)).
ADVERSE EVENT REPORTING
You must submit adverse experience reports in accordance with the adverse experience reporting requirements for licensed biological products (21 CFR 600.80) and you must submit distribution reports as described in 21 CFR 600.81. Please define, capture and submit any lack of effect events, especially those related to the treatment of gastrointestinal (GI) bleeding. These events should be reported under a separate section in the Periodic Safety Update Reports (PSURs). You should submit postmarketing adverse experience reports and distribution reports to the Office of Biostatistics and Epidemiology/HFM-210, Center for Biologics Evaluation and Research, Food and Drug Administration, 1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448. Prominently identify all adverse experience reports as described in 21 CFR 600.80. Per 21 CFR 600.2(f), please refer to http://www.fda.gov/AboutFDA/CentersOffices/CBER/ucm106001.htm for updated mailing address information.
In addition, you must submit adverse event reports for any infectious disease transmission within 15 days after learning of the event. Infectious disease transmission refers to an adverse event that involves suspected or confirmed transmission of an infectious agent, whether the recipient develops the infectious disease or only has serologic or other evidence. If an infectious disease transmission event is serious and unexpected, you must submit a 15-day "alert report" as required under 21 CFR 600.80(c)(1)(i). Infectious disease transmission events that do not meet criteria for expedited submission require periodic reports and must be submitted as individual case reports within 15 days as authorized under 21 CFR 600.80(c)(2)(i). You should submit reports for all other non-expedited adverse events under the periodic reporting requirements specified in 21 CFR 600.80(c)(2).
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.
If you have any questions, please contact the Regulatory Project Manager, Pauline Cottrell, at
Jay S. Epstein, M.D.
Office of Blood Research and Review
Center for Biologics
Evaluation and Research