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Shire pulls Replagal submission in US after clinical trial demand

Withdraws marketing application for Fabry disease treatment

Shire has been forced to withdraw its US marketing application for Fabry disease treatment Replagal after it became apparent that the FDA would require additional clinical trials.

Shares in the company started to slide this morning on the announcement that the need for additional controlled studies will cause a "significant delay" for Replagal (agalsidase alfa) in the US and would have made it possible to secure approval "only in the distant future".

The decision to withdraw the application is a blow to the Fabry disease community, which has been suffering since 2009 from shortages in Genzyme's Fabrazyme (agalsidase beta) - the only FDA-approved drug for the rare genetic disorder - as a result of manufacturing problems at the firm's Allston Landing plant.

Shire's frustration is tangible, with its official statement noting that it was encouraged by the FDA to submit an application for Replagal and that it was anticipating "a quick review process" based on discussions with the agency, which had allowed Replagal a fast-track application.

With the FDA's blessing, Shire had made supplies of Replagal available to Fabry patients in the US - free of charge via treatment access programmes - to help alleviate the shortages. Around 140 patients or 20 per cent of the US Fabry population received the drug.

Replagal has been approved in the EU for over 10 years and is marketed in 46 countries around the world, treating over 70 per cent of the Fabry patient population worldwide.

"Shire has had a close partnership with the global Fabry patient community for over 10 years, and we are extremely disappointed that we feel compelled to make this decision," commented Sylvie Gregoire, president of the company's Human Genetic Therapies unit.

Shire's loss will be Genzyme's gain, given that parent company Sanofi recently said it had started shipping the first supplies of Fabrazyme from its new plant in Framingham, Massachusetts, and would be able to get US Fabry patients back to full dosing levels this month.

Replagal sales were $475m last year, up 35 per cent on 2010, while Sanofi booked €109m from Fabrazyme, a rise of 9 per cent. Analysts have predicted that US approval would have added around $100m to Replagal's peak sales.

15th March 2012

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