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       AThis transcript has not been edited or corrected, but appears as received from the commercial transcribing service.  Accordingly, the FDA makes no representation to its accuracyY@

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         4               FOOD AND DRUG ADMINISTRATION

         5        CENTER FOR BIOLOGICS EVALUATION AND RESEARCH

         6

         7

         8

         9               BIOLOGICAL RESPONSE MODIFIERS

        10                 ADVISORY COMMITTEE (BRMAC)

        11                         Meeting 36

        12                          DAY TWO

 

        13

 

        14

 

        15

 

        16

 

        17

 

        18

 

        19

 

        20

 

        21                   Gaithersburg, Maryland

 

        22                  Friday, October 10, 2003

 

 

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                                                             2

         1     PARTICIPANTS:

 

         2        BRMAC MEMBERS:

 

         3           MAHENDRA S. RAO, Acting Chair

                     National Institute on Aging

         4

                     JONATHAN S. ALLAM

         5           Southwest Foundation for Biomedical Research

 

         6           BRUCE R. BLAZAR

                     University of Minnesota

         7

                     DAVID M. HARLAN

         8           National Institute of Diabetes and Digestive

                     and Kidney Disease

         9

                     KATHERINE A. HIGH

        10           University of Pennsylvania

 

        11           JOANNE KURTZBERG

                     Duke University Medical Center

        12

                     ALISON F. LAWTON

        13           Genzyme Corporation

 

        14           RICHARD C. MULLIGAN

                     Harvard Medical School

        15

                     ANASTASIOS A. TSIATIS

        16           North Carolina State University

 

        17           ALICE J. WOLFSON

                     Wolfson & Schlichtmann

        18

                  TEMPORARY VOTING MEMBERS:

        19

                     JAMES F. CHILDRESS

        20           University of Virginia

 

        21           LYNNE L. LEVITSKY

                     Harvard Medical School

        22

 

 

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         1     PARTICIPANTS (CONT'D):

 

         2        TEMPORARY VOTING MEMBERS (CONT'D):

 

         3           ABBEY S. MEYERS

                     National Organization for Rare Disorders

         4

                     CAROLE B. MILLER

         5           St. Agnes Healthcare

 

         6           W. MICHAEL O'FALLON

                     Mayo Clinic

         7

                     DANIEL R. SALOMON

         8           The Scripps Research Institute

 

         9           ROBERT S. SHERWIN

                     Yale University School of Medicine

        10

                     JANET H. SILVERSTEIN

        11           University of Florida College of Medicine

 

        12        CONSULTANTS:

 

        13           JOHN J. O'NEIL JR.

                     LifeScan, Inc.

        14

                     CAMILLO RICORDI

        15           University of Miami School of Medicine

 

        16        GUESTS/GUEST SPEAKERS:

 

        17           BERNARD J. HERING

                     University of Minnesota

        18

                     JAMES SHAPIRO

        19           University of Alberta

 

        20           THOMAS L. EGGERMAN

                     National Institute of Diabetes and Digestive

        21           and Kidney Diseases

 

        22

 

 

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                                                             4

         1     PARTICIPANTS (CONT'D):

 

         2        GUESTS/GUEST SPEAKERS (CONT'D):

 

         3           JAMES BURDICK

                     Health Resources and

         4           Services Administration

 

         5           FRANCISCA AGBANYO

                     Health Canada

         6

                  FOOD & DRUG ADMINISTRATION (FDA) PARTICIPANTS:

         7

                     JESSE L. GOODMAN

         8           Center for Biologics Evaluation

                     and Research

         9

                     KATHRYN CARBONE

        10           Center for Biologics Evaluation

                     and Research

        11

                     RAJ PURI

        12           Center for Biologics Evaluation

                     and Research

        13

                     CAROLYN WILSON

        14           Center for Biologics Evaluation

                     and Research

        15

                     ANDREW BYRNES

        16           Center for Biologics Evaluation

                     and Research

        17

                     NANCY MARKOWITZ

        18           Center for Biologics Evaluation

                     and Research

        19

                     STEVEN BAUER

        20           Center for Biologics Evaluation

                     and Research

        21

 

        22

 

 

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                                                             5

         1     PARTICIPANTS (CONT'D):

 

         2        FDA PARTICIPANTS (CONT'D):

 

         3           AMY ROSENBERG

                     Center for Drug Evaluation

         4           and Research (CDER)

 

         5           EMILY SHACTER

                     Center for Drug Evaluation

         6           and Research

 

         7           GAIL DAPOLITO

                     Executive Secretary

         8           Center for Biologics Evaluation

                     and Research

         9

                     ROSANNA L. HARVEY

        10           Committee Management Specialist

                     Center for Biologics Evaluation

        11           and Research

 

        12        BRMAC #36 FDA PLANNING COMMITTEE MEMBERS:

 

        13           PHILIP NOGUCHI

                     Center for Biologics Evaluation

        14           and Research

 

        15           CYNTHIA RASK

                     Center for Biologics Evaluation

        16           and Research

 

        17           DARIN WEBER

                     Center for Biologics Evaluation

        18           and Research

 

        19           DWAINE RIEVES

                     Center for Biologics Evaluation

        20           and Research

 

        21           KEITH M. WONNACOTT

                     Center for Biologics Evaluation

        22           and Research

 

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                                                            6

         1     PARTICIPANTS (CONT'D):

 

         2        BRMAC #36 FDA PLANNING COMMITTEE MEMBERS

                  (CONT'D):

         3

                     NICHOLAS I. OBIRI

         4           Center for Biologics Evaluation

                     and Research

         5

                     RICHARD McFARLAND

         6

                     STEPHEN GRANT

         7

                     SUSAN LEIBENHAUT

         8

                     JOHN ELTERMANN JR.

         9

                     JOHN FINKBOHNER

        10

                     SARAH KIM

        11

                     SUSAN ELLENBERG

        12

                     GHANSYAM GUPTA

        13

                     ROBERT MISBIN

        14

 

        15                      C O N T E N T S

 

        16     AGENDA SESSION:                            PAGE

 

        17        Current Status of Clinical Islet           7

                  Transplantation

        18

                  Allocation of Pancreata for Whole         65

        19        Organ and Islet Transplantation

 

        20        Ethical Considerations in Allogeneic     115

                  Islet Transplantation

        21

                  Clinical Development of Islet Products   151

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                                *  *  *  *  *

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         1                   P R O C E E D I N G S

 

         2                                             (8:13 a.m.)

 

         3               DR. SHAPIRO:  Good morning.  On

 

         4     behalf of Dr. Camillo Ricordi, and

 

         5     Dr. Bernhard Hering, and myself, and many

 

         6     other members involved with total islet

 

         7     transplant activity.  It is a great pleasure

 

         8     to provide this information today to the FDA

 

         9     as we consider moving forward with a

 

        10     possibility of a BLA.

 

        11               I'd like to emphasize that we come

 

        12     to you today with a long history of research

 

        13     and progress in islet transplantation over

 

        14     now three decades.  With the first

 

        15     successful islet transplantation and

 

        16     reversal of diabetes in rodent models by

 

        17     Dr. Paul Lacy and his colleagues back in the

 

        18     early 1970s.

 

        19               The success at the University of

 

        20     Minnesota with islet auto‑transplants in

 

        21     the 1970s, and in fact it's worth mentioning

 

        22     that today there are islet autografts that

 

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         1     are functional beyond 18 years maintaining

 

         2     insulin independence with no concerns

 

         3     regarding the health of those patients

 

         4     receiving islet autografts ‑‑

 

         5                    (Power plug pulled)

 

         6               DR. SHAPIRO:  As I was saying,

 

         7     the 30 years of research and then the 18

 

         8     years of success with islet

 

         9     auto‑transplants, and then the development

 

        10     of the automated method by Dr. Camillo

 

        11     Ricordi introduced and published in 1989,

 

        12     and then the first series of patients

 

        13     receiving allo transplants using the

 

        14     automated method in 1990; the success at

 

        15     Giessen and more recently in Minnesota by

 

        16     Dr. Bernhard Hering and his group, and also