Shire reports positive results from Gaucher disease drug trial

Shire has published data from a phase III clinical trial of its investigational treatment for type one Gaucher disease, which it claims to support its safety and efficacy.

Results from the study indicate that those treated using velaglucerase alfa, Shire’s enzyme replacement therapy, demonstrated immune responses which met the company’s primary targets for the trial.

These positive findings were supported further by follow-up data from an earlier five-year phase I/II study, showing that patients using the drug experienced sustained increases in haemoglobin concentration and platelet count.

Dr Pramod Mistry, professor of paediatrics and internal medicine at Yale University School of Medicine, stated that these findings could represent a significant step for sufferers of the autosomal recessive disorder.

He said: “The combined data presented today provides additional and compelling support for the long-term clinical efficacy and safety of velaglucerase alfa.”

In December 2009, the pharmaceutical company’s non-executive director Dr Michael Rosenblatt stepped down from his position at the firm after being appointed to a new role at Merck Sharp and Dohme.