In April 2000, at Easter, Anton and I went on holiday in Andalusia, Spain. I always wanted to see the beautiful Moorish architecture of the Alhambra near Granada and the Mesquita in Cordoba. When we returned home, my parents told us that Genzyme would stop the development of human alpha-glucosidasis in rabbits. For us this news was a complete shock as the Lancet had just published a convincing article that showed that enzyme replacement therapy was safe and effective in children with infantile onset Pompe disease (Van den Hout H, Reuser AJ, Vulto AG, Loonen MC, Cromme-Dijkhuis A, Van der Ploeg AT. Recombinant human α-glucosidase from rabbit milk in Pompe patients. Lancet 2000; 356:397-398). Many Dutch Pompe patients were emotional about this decision. The dutch company Pharming that we got to know so well from the beginning and that had proved to have a treatment that was effective, was now put aside without getting a real treatment in return. The treatment that would be further developed had still to be proven effective at that time
The decision to stop production of the rabbit enzyme received extensive coverage in the Dutch press
On September 28, 2001 Genzyme completed the take over of Oklahoma City-based Novazyme.
On October 30, 2001 Genzyme acquired the manufacturing facility in Geel, Belgium. In a pressrelease the following was said: ‘CAMBRIDGE, Mass.-Genzyme Corp. announced today that it has acquired certain assets of Pharming N.V., the Belgian subsidiary of the Pharming Group currently operating under a court-supervised receivership. These assets include a 70,000 square foot cGMP protein manufacturing facility currently under construction and a pilot plant that is currently used to produce transgenic human alpha-Glucosidase, both located in Geel, Belgium. The acquisition has been approved by the Commercial Court in Turnhout, the Province of Antwerp, and Genzyme's board of directors. In the near term, the acquisition of the Geel facility is intended to allow Genzyme to assume control over the production of the transgenic enzyme and secure its supply to nine patients with Pompe disease participating in the extension of a clinical trial. Genzyme has been solely funding the production of the enzyme since Pharming Group sought receivership’.
When we heard about the financial problems of Pharming. It was a shock to all of us. Who would have ever thought that the company that was developing and producing our treatment was not able to manage and continue the production? It was a real nightmare and it made clear that even when a treatment is successful, the race isn’t finalised. Patients always will be in a vulnerable position as they always will be dependent on markets, CEOs, politics, researchers, physicians, governments etc.
When Pharming had financial problems it became a difficult period for patients, physicians and the employees of Pharming. At a certain moment there wasn’t even money to pay salaries of those people who were producing the ERT in Geel, Belgium. Most of these people were young, just starting their carreer. I really was impressed with their persistence and loyalty to continue working for those 9 Pompe patients in the Netherlands and Germany who were depended on their ERT. They even continued working when they didn’t know if they would receive a salary for it. The problems of Pharming and especially the threads of the financial problems for the Pompe patients were discussed in the news.
When Genzyme finally took over the Pompe program from Pharming. The employees in Geel were taken over as well. Most of these employees stayed at Genzyme to continue producing the ERT with the genetically modified rabbits. Many of them still work there and are closely involved in the Myozyme production in the 4000 Liter bioreactors in Geel.