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Guidance to the Gaucher Community on the Management of Cerezyme Supply

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					            Guidance to the Gaucher Community
           on the Management of Cerezyme Supply
                     Temporary Conservation of Cerezyme Supply to
                Protect the Most Vulnerable Patients with Gaucher Disease




Guidance prepared by the Cerezyme Stakeholders Working Group*, 22 June 2009

Robin Ely, MD                                                           Pramod Mistry, MD, PhD
President, Medical Director                                             Yale University
National Gaucher Foundation                                             School of Medicine

Rhonda Buyers                                                           Gregory Pastores, MD
CEO, Executive Director                                                 New York University
National Gaucher Foundation
                                                                        Neal Weinreb, MD
John Barranger, MD, PhD                                                 University Research Foundation
University of Pittsburgh
Consultant, Genzyme Corporation                                         John Yee, MD, MPH
                                                                        Global Medical Affairs
Roscoe Brady, MD                                                        Genzyme Corporation
National Institutes of Health
                                                                        Kathleen Coolidge, LICSW
Gregory Grabowski, MD                                                   Patient Advocacy
Cincinnati Children's Hospital Medical Center                           Genzyme Corporation

*Please note that some individuals who participated in the Cerezyme Stakeholders Working Group are employees of
Genzyme and other individuals or their institutions or organizations receive or have received funding from Genzyme for
research, educational activities, and other purposes.




                                                        Page 1
                    Guidance to the Gaucher Community
                   on the Management of Cerezyme Supply

Background

Genzyme recently identified a virus (vesivirus 2117) in one of six bioreactors at its Allston
manufacturing facility. This virus is not known to cause disease in humans, but it can impair the
viability of the non-human CHO cells used to produce Cerezyme. Genzyme has temporarily
suspended the production of Cerezyme® (imiglucerase for injection) in order to sanitize this
facility and to restore full production as quickly as possible. This manufacturing interruption will
result in a temporary shortage in the supply of Cerezyme.

Additional lots of Cerezyme produced at the Allston facility have been tested for the presence of
vesivirus 2117. No evidence of this virus has been detected. Based on this information, the FDA
authorized Genzyme to begin shipping the Cerezyme in inventory that had completed testing.
Genzyme continues to work with the FDA to test additional lots of Cerezyme that have not yet
been released.

However, even with the release of this additional inventory, Genzyme still anticipates a supply
shortage for Cerezyme. If no changes are made in the current patterns of Cerezyme use, the
available supply is expected to “stock-out” at the end of July 2009 and the shortage is currently
expected to continue through mid-September or early October 2009 when additional Cerezyme is
anticipated to be available following the restart of production (a stock-out period of approximately
6 – 8 weeks, based on current assumptions).




Gaucher disease is heterogeneous in its clinical manifestations and progression. A temporary
interruption of Cerezyme treatment could have adverse health consequences for some of the
most vulnerable patients. For this reason, a plan is needed to protect the most vulnerable patients
by allowing them to continue treatment with Cerezyme at their current dosing regimen throughout
the period of supply shortage.

In order to accomplish this objective, a Cerezyme Stakeholders Working Group was convened to
develop and disseminate guidance recommendations to temporarily decrease the amount of
Cerezyme used by the less vulnerable patients so that the conserved supply could be used to
protect the most vulnerable patients until an adequate supply of Cerezyme is restored.




                                                Page 2
The Cerezyme Stakeholders Working Group includes leaders of the National Gaucher
Foundation (NGF, the largest Gaucher patient advocacy organization), a group of internationally-
recognized physicians with deep clinical and scientific expertise in Gaucher disease (members of
the NGF Medical Advisory Board, who provided the medical recommendations for this guidance),
and representatives from Medical Affairs and Patient Advocacy at Genzyme Corporation (who
provided background information, guidance framework and coordinated the meeting).

The Cerezyme Stakeholders Working Group embraced several principles in developing this
guidance for temporary conservation of Cerezyme supply:

    •   The guidance should be designed to protect the most vulnerable patients.

    •   The guidance should be designed to minimize risk for all other patients.

    •   The guidance should be the same irrespective of commercial or charitable access status.

    •   The guidance should be based on the best available evidence and experience.

    •   The guidance should aim for wide dissemination and compliance.

    •   The guidance should be simple to understand and practical to implement.

    •   Physicians should make the final treatment decisions regarding their patients.




                                              Page 3
Recommendations
The Cerezyme Stakeholders Working Group met via web conference on 22 June 2009 to address
two objectives:
    1. Develop a working definition of the most vulnerable patients whose Cerezyme treatment
       should not be interrupted.
    2. Formulate recommendations for temporary reductions of Cerezyme dosing for all other
       patients.

This guidance is based on the assumption that these recommendations should be applied only on
a temporary, short-term basis, effective immediately, until the period of Cerezyme shortage ends.



Group 1: Most vulnerable patients

    a. Infants, children and adolescents (≤18 years old) and patients with type 3 Gaucher
       disease should continue receiving Cerezyme according to their current dose and
       frequency, without any interruptions.

    b. Adult patients (>18 years old), with active disease progression (e.g., pulmonary
       hypertension, active skeletal disease [bone crisis and/or osteonecrosis within previous 90
       days], severe thrombocytopenia, severe anemia, or other signs or symptoms which
       would place the patient at a high risk of developing complications following a dose
       reduction, as determined by the patient’s physician) should continue Cerezyme according
       to their current dose and frequency, without any interruptions.

    c.   Pregnant women who are currently receiving Cerezyme should continue according to
         their current dose and frequency, without any interruptions, assuming the indication and
         need are clear and after a careful risk/benefit analysis has been conducted.

    d. Newly diagnosed patients who meet the criteria for Group 1 (based on [a] or [b] above)
       are recommended to initiate treatment with Cerezyme promptly.




Group 2: Less vulnerable patients

    a. Adult patients (>18 years old) without clinical evidence of active disease progression
       should reduce their dose of Cerezyme by 50% of their current dose, but not lower than
       15U/kg every 2 weeks (if currently receiving infusions every 2 weeks) or not lower than
       30U/kg every 4 weeks (if currently receiving infusions every 4 weeks).

    b. It is recommended that patients in Group 2 should have their hemoglobin level, platelet
       count, and chitotriosidase activity level (if available) measured at baseline and every 4
       weeks during the period of temporary dose reduction.

    c.   If patients in this group show signs or symptoms of clinical worsening, it is recommended
         that the Cerezyme dose be adjusted back to the dose used prior to the dose reduction.

    d. Newly diagnosed patients who do not meet the criteria for Group 1 should defer initiating
       treatment with Cerezyme until the period of Cerezyme shortage has ended.




                                              Page 4
General Guidance for All Patients

   a. The recommendations should be implemented immediately and widely in order to
      conserve an adequate supply of Cerezyme for the most vulnerable patients.

   b. The recommendations should be continued until notification by Genzyme that adequate
      supply has been restored.

   c.   At the end of this temporary period of Cerezyme shortage, it is recommended that all
        patients should resume their previously prescribed dosage regimen.

   d. The recommendations may be subject to change if the guidance is not widely adopted or
      if Cerezyme production timelines need to be revised.




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