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October 23, 2014 Approval Letter - OBIZUR

October 23, 2014 Approval Letter - OBIZUR

October 23, 2014

Our STN: BL 125512/0

Baxter Healthcare Corporation
Attention: Iraj Daizadeh, PhD
One Baxter Way
Westlake Village, CA 91362

Dear Dr. Daizadeh:

We have approved your biologics license application (BLA) for Antihemophilic Factor (Recombinant), Porcine Sequence effective this date. You are hereby authorized to introduce or deliver for introduction into interstate commerce Antihemophilic Factor (Recombinant), Porcine Sequence under your existing Department of Health and Human Services U.S. License No. 0140. Antihemophilic Factor (Recombinant), Porcine Sequence is indicated for the treatment of bleeding episodes in adults with acquired hemophilia A.

Under this license, you are approved to manufacture Antihemophilic Factor (Recombinant), Porcine Sequence drug substance at your Baxter facility in -----------(b)(4)---------. The final formulated product will be manufactured and filled at the ------------(b)(4)---------- facility in -----------------(b)(4)----------------- and labeled and packaged at the Baxter -------(b)(4)-------- facility in -----(b)(4)--------. The diluent (Sterile Water For Injection in 1 mL pre-filled syringe) will be manufactured at the ----------------(b)(4)---------------------------------------- facility in -----(b)(4)---------------- ------------------------(b)(4)--------------- facility in ----(b)(4)-------- You may label your product with the proprietary name OBIZUR and will market it in nominal potency of 500 Factor VIII units per vial.

We did not refer your application to the Food and Drug Administration 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 which would have benefited from an advisory committee discussion.

The dating period for Antihemophilic Factor (Recombinant), Porcine Sequence drug product shall be 24 months from the date of manufacture when stored at 2 - 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 or reworking is allowed without prior approval from the Agency. The dating period for your drug substance shall be ---(b)(4)--- when stored at ---(b)(4)---.

You currently are not required to submit samples of future lots of Antihemophilic Factor (Recombinant), Porcine Sequence to the Center for Biologics Evaluation and Research (CBER) for release by the Director, CBER, under 21 CFR 610.2. We will continue to monitor compliance with 21 CFR 610.1 requiring completion of tests for conformity with standards applicable to each product prior to release of each lot.

You must submit information to your BLA 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 Antihemophilic Factor (Recombinant), Porcine Sequence, or in the manufacturing facilities.

You must submit reports of biological product deviations under 21 CFR 600.14. You should identify and investigate all manufacturing deviations promptly, 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, at the following address:

Food and Drug Administration
Center for Biologics Evaluation and Research
Document Control Center
10903 New Hampshire Ave
WO71-G112
Silver Spring, MD 20993-0002

We hereby approve the draft package insert labeling submitted on October 15, 2014 and the draft carton and container labeling submitted on October 9, 2014.

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.

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 Advertising and Promotional Labeling Branch at the following address:

Food and Drug Administration
Center for Biologics Evaluation and Research
Document Control Center
10903 New Hampshire Ave
WO71-G112
Silver Spring, MD 20993-0002

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)].

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. You should submit postmarketing adverse experience reports and distribution reports to the Office of Biostatistics and Epidemiology, at the following address:

Food and Drug Administration
Center for Biologics Evaluation and Research
Document Control Center
10903 New Hampshire Ave
WO71-G112
Silver Spring, MD 20993-0002

Prominently identify all adverse experience reports as described in 21 CFR 600.80.

PEDIATRIC REQUIREMENTS

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.

POSTMARKETING COMMITMENT SUBJECT TO REPORTING REQUIREMENTS UNDER SECTION 506B

We acknowledge your written commitment as described in your letter of October 21, 2014 as outlined below:

  1. Baxter Healthcare Corporation commits to collecting additional efficacy and safety data for OBIZUR in adults with acquired hemophilia A in the Treatment Registry study under Protocol 241302, “A Non-Interventional Study of Safety and Effectiveness of Recombinant Porcine Sequence FVIII (OBIZUR) in the Treatment of Bleeding Episodes for Patients with Acquired Hemophilia A.”

    Final protocol submission date: 31 March 2015

    Study/trial completion date: 30 September 2019

    Final Report Submission date: 31 January 2020

Please submit the clinical protocol to your IND 10695, with a cross-reference letter to this BLA, STN BL 125512/0 explaining that this protocol was submitted to the IND. Submit correspondence, annual reports, and the final study report to your BLA, STN BL 125512. If the information in the final study report supports a change in the labeling, the final study report should be submitted as a supplement. We may also request a supplement if we think labeling changes are needed. Please use the following designators to label prominently all submissions, including supplements, relating to this postmarketing study commitment as appropriate:

  • Postmarketing Study Commitment Protocol
  • Postmarketing Study Correspondence
  • Postmarketing Study Commitment – Final Study Report
  • Supplement Contains Postmarketing Study Commitments – Final Study Report

Because this postmarketing study is subject to the reporting requirements of 21 CFR 601.70, you must describe the status in an annual report on postmarketing studies for this product. Label your annual report as “Annual Status Report of Postmarketing Study Commitments.” The status report for each study should include:

  • the postmarketing commitment number for this study as shown in this letter;
  • information to identify and describe the postmarketing commitment;
  • the original schedule for the commitment;
  • the status of the commitment (i.e., pending, ongoing, delayed, terminated, or submitted); and
  • an explanation of the status including, for clinical studies, the patient accrual rate (i.e., number enrolled to date and the total planned enrollment).

As described in 21 CFR 601.70(e), we may publicly disclose information regarding this postmarketing study on our Web site (http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIVCommitments/default.htm). Please refer to the February 2006 Guidance for Industry: Reports on the Status of Postmarketing Studies – Implementation of Section 130 of the Food and Drug Administration Modernization Act of 1997 (see http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM080569.pdf) for further information.

PDUFA V APPLICANT INTERVIEW

FDA has contracted with Eastern Research Group, Inc. (ERG) to conduct an independent interim and final assessment of the Program for Enhanced Review Transparency and Communication for NME NDAs and Original BLAs under PDUFA V (‘the Program’). The PDUFA V Commitment Letter states that these assessments will include interviews with applicants following FDA action on applications reviewed in the Program. For this purpose, first-cycle actions include approvals, complete responses, and withdrawals after filing. The purpose of the interview is to better understand applicant experiences with the Program and its ability to improve transparency and communication during FDA review.

ERG will contact you to schedule a PDUFA V applicant interview and provide specifics about the interview process. Your responses during the interview will be confidential with respect to the FDA review team. ERG has signed a non-disclosure agreement and will not disclose any identifying information to anyone outside their project team. They will report only anonymized results and findings in the interim and final assessments. Members of the FDA review team will be interviewed by ERG separately. While your participation in the interview is voluntary, your feedback will be helpful to these assessments.

Sincerely yours,

Jay S. Epstein, MD
Director
Office of Blood Research and Review
Center for Biologics Evaluation and Research

Enclosure:
Final Approved Labeling