Shire plc (LSE: SHP, NASDAQ: SHPGY, TSX: SHQ) announced that Japan’s Ministry of Health, Labour and Welfare has approved ELAPRASE, a human enzyme replacement therapy for the treatment of Hunter syndrome, for sale and marketing in Japan. Since its U.S. approval in July 2006, ELAPRASE is now approved for marketing and commercial distribution in 34 countries worldwide.
Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, life-threatening genetic condition mainly affecting males that results from a deficiency in 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, developed by Shire Human Genetic Therapies, Inc. (HGT) is the first and only enzyme replacement therapy approved for people suffering from Hunter syndrome. This medicine is given as a weekly infusion and is designed to replace the deficient iduronate-2-sulfatase enzyme.
As part of an agreement with Genzyme Corporation, Genzyme will manage sales and distribution of ELAPRASE in Japan.
“The approval of ELAPRASE in Japan is another important step in bringing this much-needed treatment to Hunter syndrome patients around the world,” said Sylvie Grégoire, president of Shire HGT, the Shire business focused on genetic diseases. “We greatly appreciate the commitment and determination invested by the Japanese patients and their families who temporarily moved to the United States to participate in the ELAPRASE pivotal trial. Their participation in the trial facilitated a rapid approval of ELAPRASE, allowing it to now be available to Hunter syndrome patients in Japan.”