IPA - International Pompe Association

Genzyme/Novazyme statement on future of patients currently in trials

Genzyme/Novazyme statement on patients invovled in transgenic enzyme trials

Posted to GSDNet by Paul Kaplan on 22 August 2001

Ultimately, it is Genzyme’s goal to transition all of the patients currently receiving transgenic alpha-glucosidase to the CHO-derived derived drug. I assure you that Genzyme will do everything possible to make certain that the nine patients currently receiving transgenic drug continue to receive therapy. Genzyme has now provided the necessary financial guarantees to allow Pharming to continue the uninterrupted production of the transgenic enzyme. The safety and efficacy of the transition to the CHO product is of utmost importance.

Preliminary studies conducted to date with transgenic and CHO enzymes suggest that encouraging results have occurred. However, having acknowledged this Genzyme/Pharming has decided to pursue development of a CHO based therapy for Pompe disease.

Let me explain why. Genzyme/Pharming believes that there have not yet been enough patients treated with either drug to allow a regulatory agency to evaluate the safety and effectiveness of either drug for approval. Our previously stated intention switch to developing the CHO-based product was based on manufacturing considerations. Genzyme/Pharming believes that alpha-glucosidase produced from CHO cells is the solution to delivering a Pompe therapy quickly to as many patients as possible world-wide. CHO is a proven technology that has been used repeatedly to produce large quantities of protein drugs that have been routinely approved by regulatory authorities throughout the world.

As we develop therapies for Pompe disease and other lysosomal storage diseases, we will continue to invest in technologies that will improve on our existing products and products in development. Therefore, Genzyme is making continuous investments in enzyme replacement technology, and hence, our investment in Novazyme, with its specific technology, and in gene and cell therapy efforts around the world.

Genzyme’s goal is to support those initiatives that ultimately will bring the best possible product to as many patients as possible and to do so as quickly as possible. In order to do this effectively we will take into account data and scientific knowledge gathered from all sources, including ongoing and future assessments of patients receiving drug from all sources. This will enable those involved in all phases of research into Pompe disease to build upon all available data and thus work towards the most effective, rapidly available, treatment.

We look forward to the September AMDA meeting in San Antonio to discuss clinical and scientific subjects among patients and clinical experts.