Shire withdraws FDA application for Replagal for Fabry Disease

Shire has withdrawn its Biologics License Application (BLA) for Replagal (agalsidase alfa) with the FDA. Shire has been in ongoing dialogue with the FDA since the supply shortage of the only USA approved treatment for Fabry disease. In 2009, and again in 2011, the FDA encouraged Shire to submit an application for the approval of Replagal. Recent interactions with the FDA have led the Company to believe that the agency will require additional controlled trials for approval. No concerns over the product�s safety profile were raised by the FDA. Shire has concluded that the likely additional studies would cause a significant delay, and an approval of Replagal for USA patients would only be possible in the distant future. Shire has therefore decided to withdraw its BLA. Replagal has been approved in the European Union for over 10 years and is marketed in 46 countries around the world, treating over 70% of the Fabry patient population worldwide.