Shire plc (LSE: SHP, Nasdaq: SHPGY), the global specialty biopharmaceutical company, presented new data for Replagal® (agalsidase alfa) in patients with Fabry disease, and VPRIV® (velaglucerase alfa for injection) in patients with type 1 Gaucher disease, at the 2011 American College of Medical Genetics' (ACMG) Annual Clinical Genetics Meeting, held in Vancouver, BC, from March 16-20, 2011.
Data from clinical programs evaluating the safety of switching from Fabrazyme® (agalsidase beta) to Replagal and long-term safety and efficacy of switching from Cerezyme® (imiglucerase) to VPRIV were presented.
Preliminary data from Shire's Replagal study 059, an ongoing, multicenter, open-label clinical study, was presented for the first time. This data, following 3 months of treatment, suggests that switching from Fabrazyme to Replagal 0.2 mg/kg every other week is generally well tolerated. This ongoing study includes 70 Fabry patients in the US under Shire's treatment protocol who were switched to Replagal during the continuing Fabrazyme supply shortage. The safety events observed in this study were similar to those seen historically in patients treated with Replagal.
Data from the 52 patients in Shire's VPRIV study 044, the extension study for studies 034 and 039, were presented. Data from study 058, Shire's treatment protocol in the US, in 205 type 1 Gaucher patients who switched from Cerezyme were also presented. Taken together, these studies include data from ongoing treatment in more than 250 patients, representing the largest and most comprehensive set of studies to date for Gaucher disease. This collection of long-term data (up to 2 years) adds to the growing body of clinical evidence which supports the use of VPRIV in patients who have previously been treated with imiglucerase.
"The data presented here reinforces our confidence in the treatments that Shire has developed for Fabry and Gaucher diseases. I am pleased that Shire has been able to continue supporting the Fabry and Gaucher patient communities," commented Dr. Paul M. Fernhoff, Division of Medical Genetics of the Emory University School of Medicine.
Replagal and VPRIV were developed by Shire using its proprietary gene activation technology in human cell lines. Shire has utilized this technology to manufacture enzyme replacement therapies for nearly two decades.
"We are pleased to continue our commitment to support patients and healthcare providers who are dedicated to treating rare diseases. The data presented at ACMG strengthens the body of evidence for Replagal and VPRIV as treatments that may benefit Fabry and Gaucher patients," said Sylvie Grégoire, President of Shire HGT. "Shire has invested more than $200 million on enhancing capacity and technology in order to ensure safe and consistent drug supplies, and we anticipate the first approval of the new Lexington manufacturing facility at the end of 2011."