Genzyme Corp. announced that it has received approval to market Fabrazyme (agalsidase beta) in Japan for the treatment of Fabry disease. The company expects to launch Fabrazyme as soon as the Japanese Ministry of Health, Labor and Welfare establishes a local market price for the product.
Genzyme estimates there are several hundred patients in Japan with Fabry disease. The company completed a study of Fabrazyme in Japan in 2001.
"The approval of Fabrazyme has been eagerly anticipated by many Japanese physicians, Fabry patients and their families," said Yoshikatsu Eto, professor and chairman of the Department of Pediatrics at the Tokyo Jikei University School of Medicine and principal investigator for Genzyme's study of Fabrazyme in Japan.
"We are pleased to bring this therapy to patients who suffer from this serious and life-threatening disease," said Sandford Smith, president of Genzyme's international group. "We anticipate that Fabrazyme will be adopted quickly by physicians who care for Fabry patients at multiple centres in Japan."
Fabry disease is a genetic disorder caused by an enzyme deficiency. The disease causes certain fats to accumulate in the blood vessels over many years, leading to the involvement of various tissues and organs in the body, including the kidneys and heart, which can result in organ failure and stroke. People with Fabry disease often must cope with significant pain and disability and typically have a shortened life span. The disease affects approximately 5,000 people worldwide.