The European Commission has granted Shire marketing authorization for VPRIV® (velaglucerase alfa) for the treatment of type 1 Gaucher disease. This enzyme replacement therapy (ERT) is derived from a human cell line and has been given orphan drug status through the centralized procedure, making it available in 30 countries across Europe.
This approval was based on Shire's clinical development program, which evaluated over 100 Gaucher patients at 24 sites in 10 countries. Shire reports that all studies met their primary endpoints. The safety and efficacy of velaglucerase alfa was assessed in adults and children aged four years and older via a Phase III program that included Gaucher patients who switched to velaglucerase alfa after being treated with imiglucerase as well as naive patients.
VPRIV is made using Shire's gene-activation technology in a human cell line. It has the exact human amino acid sequence as the endogenous glucocerebrosidase enzyme and also has a human glycosylation pattern.
The drug has been available in Europe under early-access programs. It was approved in the U.S. in February, at which time Shire said that it would price VPRIV 15% lower than Genzyme’s Cerezyme.
Second-quarter sales of Cerezyme were $138.7 million compared with $298.1 million in the second quarter of last year. Cerezyme was shipped at 50% of demand due to supply constraints that resulted from viral contamination at Genzyme’s Allston Landing production plant. Cerezyme revenue is expected to be $725 million–$775 million by the end of 2010.
In July Genzyme reported that production of Cerezyme had been brought in line with historical averages and that it would begin increasing shipments in August. The company expects that patients will be able to begin increasing their doses in September and return to normal dosing in the fourth quarter.