Shire Plc (SHPGY) Wednesday announced data from a six-month study indicating that Fabry disease patients switching to its Replagal tolerated the change well. The Dublin, Ireland-based company also reported sustained efficacy and safety in patients with type 1 Gaucher disease who had switched to its Vpriv therapy.
The results are being presented at the 2011 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism or SSIEM held from August 30 - September 2 in Geneva, Switzerland.
Rohrbach of University Children's Hospital in Zurich, said," Patients and physicians want to be reassured that the product they are switching to is not only readily available - but also generally well tolerated. The data presented at SSIEM provides us with that evidence."
Shire reported six-month data from its HGT-REP-059 study suggesting that Replagal at its licensed dose of 0.2 mg/kg every other week is well tolerated by Fabry patients switching from Fabrazyme.
Replagal is the brand name for agalsidase alfa while Fabrazyme is the brand name for agalsidase beta. Fabry disease is an inherited disorder that results from the buildup of a particular type of fat in the body's cells.
The ongoing study was established to provide U.S. Fabry patients with access to Replagal during the Fabrazyme supply shortage. According to Shire, more than 2,600 patients are now being treated with Replagal globally, representing more than 75 percent of the treated Fabry patients worldwide.
Shire reported long-term data of Gaucher's disease patients switching to its Vpriv from Cerezyme. Vpriv is the trade name for velaglucerase alfa while Cerezyme is the trade name for imiglucerase.
Vpriv is for patients who are treatment-naive as well as patients who have previously been treated with imiglucerase. Cerezyme is made by Sanofi-aventis' (SNY) subsidiary Genzyme Corp. Gaucher's disease is a genetic disease in which a fatty substance accumulates in cells and certain organs.
In additional data from Shire's long-term open-label, Phase I/II TKT025 extension study of Vpriv, the company reported continued improvement of bone mineral density or BMD.
Shire said the data showed clinically meaningful and statistically significant improvements in lumbar spine BMD which were observed as early as 24 months and femoral neck BMD as early as 33 months in patients on Vpriv, during year three of therapy and despite significant baseline skeletal pathology.
Shire also presented data from several other clinical studies that demonstrate the continued sustained efficacy and safety profile of Vpriv in patients switching from Cerezyme.
In February 2010, Vpriv was approved by the U.S. FDA as a hydrolytic lysosomal glucocerebroside-specific indicated for long-term enzyme replacement therapy for adult and pediatric patients with type 1 Gaucher disease.
SHPGY is currently trading at $97.85, up $2.66 or 2.79%, on the Nasdaq. Over the past year, the stock traded in a range of $65.77 - $105.53.
For comments and feedback contact: editorial@rttnews.com
April 10, 2026 16:21 ET Inflation data from the U.S. was the main data event this week as the conflict in the Middle East continue. The minutes of the latest Fed policy session and the survey data on the services sector also made headlines. In Europe, manufacturing orders data from Germany was in focus. Price data from China drew attention in Asia.