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Accelerated assessment for Gaucher disease

Shire has been granted an accelerated assessment by CHMP for velaglucerase alfa to treat type 1 Gaucher disease

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

This is the third marketing application that has been submitted for velaglucerase alfa, the company's enzyme replacement therapy, with previous submissions in the US and Canada.

Based on a global supply shortage of the currently approved and marketed treatment for patients with Gaucher disease, and positive results from all velaglucerase alfa phase III trials, the CHMP has accepted the company's request for an accelerated assessment of the velaglucerase alfa MAA. The MAA review is due to start in December and under accelerated assessment, should take 150 days rather than 210 days.  

"Gaucher disease is a debilitating condition and the continuing imiglucerase supply shortage has had a significant impact on patients who have lacked an alternative supply of enzyme therapy," said Dr Timothy Cox, professor of medicine at the UK's University of Cambridge and the founder of the National Centre for the Treatment of Gaucher disease at Addenbrooke's Hospital. "Shire's partnership with health regulators and physicians to devise and implement expanded access programmes for velaglucerase alfa is greatly appreciated by treating physicians and the Gaucher community at large. We welcome the news of the submission of the velaglucerase alfa MAA in Europe."

In Europe and other countries outside the US, patients continue to receive velaglucerase alfa through pre-approval access programmes developed in partnership with national and regional authorities and designed to address the continuing supply shortage. In the US, patients are enrolled in an FDA-approved treatment protocol.

Gaucher disease is an autosomal recessive disorder that causes skeletal abnormalities and deformities as well as bone pain.

Shire's velaglucerase alfa programme included the largest and most comprehensive set of phase III clinical trials conducted for Gaucher disease in which over 100 patients at 24 sites in 10 countries around the world have participated.

25th November 2009

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