IPA /Genzyme Joint Pompe Program Update, August 2004

IPA /Genzyme Joint Pompe Program Update, August 2004



Infantile-Onset Expanded Access Program

Enrollment in the Infantile Expanded Access Program is currently on-going. Physicians who may have an infantile-onset Pompe patient should contact Genzyme Medical Information. Information for enrollment will be reviewed on a case-by-case basis. The Infantile-Onset EAP was initiated in October 2003. Infantile-onset patients have been enrolled in the program in regions around the world, and some patients have begun receiving investigational enzyme. Patients continue to be actively screened and the process for initiating sites and receiving IRB and ethics committee approval is also ongoing.

The inclusion/exclusion criteria for this program are posted on http://clinicaltrials.gov, the National Institutes of Health website. Eligibility for this program is based primarily on an onset of symptoms during a childs first year of life as well as a documented GAA deficiency. However, regulatory environments are different around the world and therefore, the inclusion/exclusion criteria may vary by region. The most expeditious way to have any questions regarding patient eligibility answered will be for a treating physician to contact Genzyme Medical Information, either in the US or Europe.

Late-Onset Expanded Access Program

There have been some very positive changes in the scope of this program. Genzyme has enrolled late-onset patients from the US, Europe, Australia, Canada, and Taiwan into the Expanded Access Program, and some of these "first round" patients have begun receiving infusions. There were also individuals who met the enrollment criteria, but who were not enrolled in the program initially; Genzyme has now begun the process of enrolling these patients in a "second round". This process may take several months and may be dependent on the resources available in the patients region, as well the treating physicians familiarity with enzyme replacement therapy.

The time that is needed for an individual patient to begin receiving investigational enzyme, after they are approved for enrollment in this program, can be quite variable. It can be influenced by the training needs of the treating physician, nurses, pharmacists, study coordinators and many others who may be involved with the care of the Expanded Access patients. In addition, it takes time to collect and review the information from the sites. Finally, it requires effort to obtain country specific and institutional regulatory approvals necessary to treat the eligible patients with this investigational enzyme. For example, some sites have obtained institutional approval in one month and in other cases it has taken 3-4 months or longer.

The priority is to ensure that the process of initiating the "first round" patients on therapy is completed as rapidly as possible. However, due to the variability across regions and of all the contributing factors, it is possible that this process could be completed for a "second round" patient before a "first round" patient.

At this time, Genzyme is not actively screening for additional late-onset patients, but will retain physician contact information in the event that additional patients can be screened in the future

If you or your physician would like more information about Genzymes Expanded Access Program, please contact Genzyme Medical Information. The email address in the United States is medinfo@genzyme.com and the telephone number is 800-745-4447 (press 2). In Europe, the email address is eumedinfo@genzyme.com and the telephone number is 31-35-699-1499, or you may contact your local Genzyme office. If you are calling from outside the US or Europe, please call 617-768-9000.

For more information from Genzyme about Pompe disease, please visit their Pompe Community website www.pompe.com.