The product is the first treatment ever approved for Pompe disease and one of the first for an inherited muscle disorder.
“This is an extraordinary moment for Pompe patients and their families,” said Henri Termeer, chairman and CEO of Genzyme. “The effort to develop Myozyme has required the enormous commitment of many people throughout Genzyme and across the Pompe community, who have worked with a great sense of urgency and have overcome tremendous challenges.”
Myozyme has received orphan medicinal product designation in Europe. The orphan medicinal products regulation is designed to encourage the development of treatments for rare disorders such as Pompe disease, for which no therapies have existed previously.
Pompe disease manifests as a broad spectrum of clinical symptoms. All patients typically experience progressive muscle weakness and breathing difficulty, but the rate of disease progression can vary widely depending on the age of onset and the extent of organ involvement.