Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the European Commission has granted marketing authorisation for VPRIV(R) (velaglucerase alfa), a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of type 1 Gaucher disease. VPRIV has been authorized as an orphan medicine through the Centralised Procedure, making it available in 30 countries across Europe.
This approval was based on data from Shire's velaglucerase alfa clinical development programme which represents the largest and most comprehensive clinical data set supporting registration for an ERT for type 1 Gaucher disease. In total, over 100 Gaucher patients at 24 sites in 10 countries around the world participated in the clinical studies, all of which met their primary endpoints.
"Gaucher disease is a rare and often debilitating condition," said Professor Tim Cox from the Department of Medicine, University of Cambridge, and founder of the largest U.K. Gaucher clinic at Addenbrooke's Hospital. "The European approval of VPRIV is important in that we now have an alternative, licensed therapeutic enzyme. For type 1 patients the availability of VPRIV provides further opportunities to individualise treatment options for this complex disorder."
Across Europe, hundreds of type 1 Gaucher patients have been receiving velaglucerase alfa through early access programmes, developed in partnership with national authorities, Gaucher expert physicians and patient associations. Globally there are over 850 patients on velaglucerase alfa and demand continues to be strong. As a result, Shire has implemented a program to carefully monitor demand and manage requests from physicians and patients in order to ensure long-term, uninterrupted treatment with VPRIV.
"The Marketing Authorisation for VPRIV in the EU is an important milestone for Shire," said Sylvie Gregoire, President, Shire Human Genetic Therapies. "Our efforts to accelerate our manufacturing, clinical and regulatory timelines have resulted in VPRIV's approval in Europe and the US months ahead of schedule."