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Positive results for Shire's Gaucher trial

Shire has reported positive results from its first phase III study of the company's treatment for Type 1 Gaucher disease

Speciality biopharmaceutical company Shire has reported positive results from its first phase III study into the safety and efficacy of velaglucerase alfa, its investigational enzyme replacement therapy for the treatment of Type 1 Gaucher disease.

Additional data from a paediatric subgroup of this study along with five-year follow-up results from a long-term phase I/II extension study conducted in adults were also reported.

The phase III study (known as TKT032) was a 12-month, randomised, double-blind, parallel-group global study in 25 treatment-naïve patients aged two years and older. The trial evaluated velaglucerase alfa at two different doses.

Patients were eligible to participate in the study if they presented with disease-related anaemia and had at least one of the clinical manifestations of Gaucher disease: thrombocytopenia, moderate splenomegaly or a readily palpable enlarged liver.

"The combined data presented today provides additional and compelling support for the long-term clinical efficacy and safety of velaglucerase alfa," said Dr Pramod Mistry, Professor of paediatrics and internal medicine at Yale University School of Medicine. "The Gaucher community is very fortunate to have velaglucerase alfa as an option for patients."

Velaglucerase alfa is an investigational enzyme replacement therapy for Type 1 Gaucher disease. Velaglucerase alfa is made using Shire's gene-activation technology, in a human cell line.

In November 2009, the Committee for Medicinal Products for Human Use (CHMP) granted an accelerated assessment of Shire's European marketing authorisation application (MAA) for velaglucerase alfa to treat type 1 Gaucher disease.

Shire is likely to face some competition in this therapy area – In December, Pfizer announced it had entered into a deal with Israeli biotech company Protalix Biotherapeutics to develop and commercialise a potential treatment for the rare genetic disorder. Earlier this month, GlaxoSmithKline announced the launch of a rare diseases unit, which will focus on the development of medicines for rare diseases, including Gaucher disease.

11th February 2010

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