The USFDA has granted approval to Shire plc (NASDAQ: SHPGY)’s ELAPRASE™, a human enzyme replacement therapy for the treatment of Hunter syndrome.
Hunter Syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, life-threatening genetic condition that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase; without which, cellular waste products accumulate in tissues and organs, which then begin to malfunction.
ELAPRASE™, the first and only treatment approved for Hunter syndrome sufferers is given as weekly infusions, replacing the missing enzyme that Hunter syndrome patients fail to produce in sufficient quantities.
“The FDA approval of ELAPRASE marks a significant milestone in Shire’s effort to provide meaningful treatments for patients suffering from genetic diseases,” said Matthew Emmens, chief executive officer of Shire.
“A hallmark of the ELAPRASE program is the commitment demonstrated by patients and families, investigators and Shire employees involved in the development effort. We look forward to making ELAPRASE available to patients in the coming weeks.”
In the next 30 days, Shire plc will launch ELAPRASE™ in the United States.
More information about ELAPRASE and Hunter syndrome can be found at the ELAPRASE™ and Hunter Patients websites, respectively, or through OnePath SM at 1 (866) 888-0660.
Read more at Shire plc.
Comment Preview