LONDON Shire's Gaucher disease drug Vpriv significantly improved lumbar spine bone density, while Sanofi's Cerezyme did not, according to results from a head-to-head clinical trial presented on Thursday.
The findings from the Shire-sponsored study, made public at a European Working Group on Gaucher Disease in Paris, may help Shire differentiate its product from Cerezyme, which is made by Sanofi's Genzyme unit.
Gaucher disease is a rare genetic disorder affecting some 10,000 patients worldwide. Patients with the disease are deficient in an enzyme that breaks down a certain type of fat, leading to potentially life-threatening organ damage and bone problems.
For many years Genzyme dominated the Gaucher market with its drug Cerezyme, at one point the most expensive drug in the world costing more than $200,000 a year.
But competition is now increasing. In addition to Vpriv, there is also another Gaucher drug called Elelyso from Protalix Biotherapeutics and Pfizer, which was approved by U.S. regulators in May but was rebuffed in Europe last week.
Ari Zimran of the Hebrew University and Hadassah Medical School, Jerusalem, said the study results showed Vpriv was effective in treating key markers of Gaucher-related bone disease.
Clinically and statistically significant improvements in bone measurements were seen at nine months of treatment with Vpriv but not with Cerezyme.
(Reporting by Ben Hirschler)