Summary of IPA/Genzyme Teleconference from September 24, 2002

Category: Archive
September 23, 2002

Baarn, October 2nd 2002

Dear IPA members,

Herewith I would like to inform you about the IPA/Genzyme telephone conference of September 24 2002.

We discussed issues concerning the production, the transition, new trials and the meeting with the Erasmus University, VSN, Parents, patients and Genzyme. These issues will also be addressed in the presentation Genzyme will give on October the 12th at the AGSD meeting in Nottingham. Genzyme will give an update of the Pompe program and conduct a patient question and answer session. On October the 13th Genzyme representatives will attend the afternoon workshops of the IPA General Member meeting and discuss with the IPA representatives the Pompe program, and future collaboration between IPA and Genzyme.
The CHO product

First I would like to mention that Genzyme decided to turn to an internally developed CHO cell rhGAA, and to manufacture the product themselves. Genzyme has considerable experience with CHO-based manufacturing (Cerezyme, Fabrazyme, Thyrogen). Based on current experience, the Genzyme developed CHO rhGAA process is expected to provide a reliable clinical and commercial supply. In addition Genzyme expects to be able to scale-up this process as necessary to accommodate capacity needs as they arise.

New studies

Genzyme has a meeting scheduled to discuss clinical protocols for two new studies with the FDA in mid-October. During this meeting Genzyme hopes to receive input from the FDA on their new protocols. Genzyme’s proposed studies will focus on the most severe infantile forms of the disease and will utilize the Genzyme CHO cell-derived rhGAA. These studies are currently envisioned to begin sometime before the end of the year.

The start dates will depend primarily on the availability of material and whether Genzyme receives necessary regulatory and trial site clearance to begin the studies. Genzyme is continuing with their preparations to submit an IND (Investigational New Drug) application to the FDA for their internally manufactured CHO cell-derived rhGAA.

The exact number of participants and the final inclusion criteria will not be finalized until the regulatory authorities have reviewed the study design.

Clinical trial site selection has not been finalized. Site selection criteria include issues such as finding investigators who have experience in conducting complex clinical trials and have the necessary facilities and resources to conduct the studies. In addition, each individual site’s IRB (Institutional Review Board) or EC (Ethics Committee) must approve the study before it can begin. Various sites in the US, Europe and Asia are currently under consideration.


Genzyme informed us that over the summer they made significant progress in addressing the need to increase manufacturing capacity for CHO cell-derived rhGAA. Genzyme has made modifications to the production of CHO cell-derived rhGAA and added new bioreactors as part of its continuing effort to address issues surrounding increasing supply.

Meeting in Rotterdam

On September 18, 2002, the Dutch patient organization, VSN, held an in-depth meeting between Genzyme, the treating physicians at Erasmus MC the parents of the patients and patients receiving the transgenic enzyme derived from rabbits’ milk. It was a very constructive meeting. The Erasmus MC expressed their confidence in the efforts of Genzyme based on the information that was given.


This past summer patients were transitioned from the transgenically derived enzyme to the CHO cell-derived enzyme. Thus far, they had a smooth transition, with similar tolerance as with transgenic infusions. Patients will be followed closely by a group of treating physicians and Genzyme for safety and efficacy.

However, Genzyme guaranteed on July 26th that the three infantile patients in the Netherlands would continue to be treated with the transgenic enzyme until the spring of 2003 when the supply of transgenic enzyme is depleted.

In conclusion

At this moment enzyme-replacement therapy is a new experimental therapy. This will bring us uncertainties now and in the future. The therapy is not available for all the Pompe patients worldwide. First the therapy has to be approved and the CHO cell-derived enzyme has to be registrated as a medicine and there has to be enough supply. Both processes take time. Valuable time. However we hope that the information above gives you an idea about the ongoing process of development of enzyme replacement therapy for people with Pompe´s disease.

With warmest regards,

Ria Broekgaarden
Secretary-IPA board

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