Genzyme receives Japanese marketing approval for Elaprase

Genzyme Corp. has announced that it has received approval to market Elaprase (idursulfase) in Japan for the treatment of Hunter syndrome.

Elaprase is an enzyme replacement therapy developed by Shire Human Genetic Therapies Inc., and Genzyme is commercializing the product in Japan and other Asia Pacific countries under an agreement with Shire. Genzyme intends to launch Elaprase this quarter, following reimbursement approval.

Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, life-threatening genetic condition mainly affecting males that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Without this enzyme, cellular waste products accumulate in tissues and organs which then begin to malfunction, leading to severe clinical complications and early mortality.

Elaprase is the fifth enzyme replacement therapy that Genzyme has introduced in Japan. The company also markets Cerezyme(R) (imiglucerase for injection) for Type 1 Gaucher disease; Fabrazyme(R) (agalsidase beta) for Fabry disease; Aldurazyme(R) (laronidase) for MPS I; and Myozyme(R) (alglucosidase alfa) for Pompe disease.

"Our deep experience in Japan in the area of lysosomal storage disorders will enable us to make Elaprase available to all patients who need treatment as quickly as possible," said David Meeker, M.D., president of Genzyme's Lysosomal Storage Disorders business unit. "We anticipate that Elaprase will become an increasingly significant contributor for us as we introduce the product across the region."

Genzyme is currently working to obtain marketing approval for Elaprase in several additional countries. The company anticipates approval in Australia by the end of this year and in Taiwan and South Korea next year.

Under its agreement with Shire, Genzyme will record product sales in the region and allocate approximately one-third of net sales to Shire. Genzyme will make a $3 million milestone payment to Shire in recognition of Japanese marketing approval, and it will make a $4 million milestone payment upon reimbursement approval.

Genzyme established commercial operations in Japan twenty years ago and currently employs more than 100 people in the country. Genzyme was the first non-Japanese biotechnology company to introduce therapeutic products in Japan without Japanese partners. The company today also markets Seprafilm(R) adhesion barrier and offers genetic testing services.