IPA - International Pompe Association

Genzyme Acts to Ensure Continued Treatment of Pompe Patients

Genzyme Acts to Ensure Continued Treatment of Pompe Patients

CAMBRIDGE, Mass—Genzyme Corp. announced today that it will fund production of the transgenic enzyme being used to treat nine patients with Pompe disease until they can be transitioned to a form of the enzyme derived from CHO cells, as planned. Genzyme took this action following the decision by its partner Pharming Group N.V. to file for receivership. Genzyme has asked for a commitment from Pharming to continue to produce the enzyme and to supply it to the hospitals involved in the treatment of these patients.

The transgenic enzyme is manufactured by Pharming, and under a joint venture agreement, Genzyme and Pharming had shared production costs equally. The nine patients previously took part in clinical trials sponsored by the companies and have remained on therapy following the conclusion of those trials.

“The welfare of the patients currently undergoing enzyme replacement therapy with the transgenic product is of utmost concern to Genzyme,” said Jan van Heek, executive vice president of Genzyme Corp.

Mr. van Heek added that once Genzyme recently became aware of Pharming’s financial situation, it initiated exhaustive discussions with the company to propose a solution that was in the best interests of patients. Genzyme’s offer to acquire Pharming’s 50 percent interest in the two joint ventures the companies formed to develop a treatment for Pompe disease was not accepted by Pharming’s board of directors. Discussions continue to resolve matters related to the joint ventures.

Genzyme and Pharming began working in partnership in 1998. The companies jointly sponsored clinical studies of transgenic human alpha-Glucosidase at the Sophia Children’s Hospital in Rotterdam, The Netherlands, and similar studies at the University Hospital of Essen, Germany. In April 2000, the companies switched the focus of their development efforts to a form of human alpha-Glucosidase derived from CHO cells. A Phase 2 clinical trial of this product is currently underway at medical centers in Europe and the United States.

Given the seriousness of Pharming’s present financial situation and the need to move forward quickly with the development of a treatment for Pompe patients, Genzyme believes it has no choice but to take over full operational and financial responsibility for the development of the CHO-cell product. As a result, Genzyme today terminated its CHO-cell joint venture agreement with Pharming in accordance with the agreement’s provisions, based on Pharming’s failure to make ongoing payments to fund the program. The joint venture for the transgenic product remains in place.

“Dealings with Pharming and its trustees have led us to conclude that assuming full responsibility for the program is the only choice to protect and advance the interests of Pompe patients,” said Mr. van Heek. “Pharming and its trustees need to focus on the survival and financial restructuring of Pharming. We need to devote our full attention to the needs of Pompe patients.”

Genzyme is committed to developing an approved treatment for patients with Pompe disease around the world, and will continue to work diligently toward reaching that goal. The company recently announced its intention to acquire Novazyme Pharmaceuticals Inc., whose lead product candidate is NZ-1001, an enzyme replacement therapy for Pompe disease that is on track to enter clinical trials in the very near future. Genzyme will move forward aggressively with the development of NZ-1001, while it simultaneously completes the ongoing Phase 2 clinical trial of the CHO-cell-derived enzyme replacement therapy.

Over the course of their three-year partnership, Genzyme has taken steps to provide support to Pharming. Genzyme was an early investor in the company. In addition, to help fund Pharming’s portion of the initial licensing payment for the CHO-cell product, Genzyme provided Pharming with a loan of $10 million in exchange for a convertible note.

Genzyme Corporation is a U.S. based biotechnology company that develops and markets products and services designed to address unmet medical needs. Genzyme’s European headquarters is located in Naarden, The Netherlands.

This press release contains forward-looking statements, including statements about: Genzyme’s plans with respect to the transgenic enzyme product and other potential therapies for Pompe disease; the anticipated acquisition of Novazyme; the potential development of products using Novazyme’s technology and potential indications thereof; and plans concerning clinical trials. Actual results may materially differ due to numerous factors, including without limitation: Pharming’s ability to continue production of the transgenic enzyme and to do so without interruption, in a timely manner and in sufficient quantities; conditions in the financial markets relevant to the proposed acquisition; the satisfaction of the conditions to closing the proposed acquisition; the risks generally associated with acquisitions; the actual timing and results of pre-clinical and clinical studies; the efficacy and safety of products; the content and timing of submissions to and decisions by regulatory authorities; and the risks and uncertainties described in Genzyme’s reports filed with the U.S. Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation Exhibit 99.2 to Genzyme’s Annual Report on Form 10-K for the year ended December 31, 2000, as amended.

Note to editors: Genzyme representatives are available to speak with the media about the Pharming situation. Please contact Bo Piela of Genzyme at 1-617-252-7785 to arrange an interview.