March 14, 2012
Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that it has withdrawn its Biologics License Application (BLA) for REPLAGAL
(agalsidase alfa) with the US Food and Drug Administration (FDA).
Shire has been in ongoing dialogue with the FDA since the supply shortage of the only US approved treatment for Fabry disease. In 2009, and again in 2011, the FDA encouraged Shire to submit an application for the approval of REPLAGAL. The information in the application included relevant updates such as manufacturing and open long-term clinical trial data. These discussions led the Company to file a BLA last November in anticipation of a quick review process and eventual approval - allowing Shire to supply more US patients with a therapy they desperately needed at the time.
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 US patients would only be possible in the distant future. Shire has therefore decided to withdraw its BLA.
"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," said Sylvie Grégoire, President, Shire HGT.
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. Over 1000 patients have switched to REPLAGAL since the beginning of the supply shortage in mid-2009. Shire has been providing REPLAGAL free of charge to around 140 US patients - about 20% of the treated US patients - through treatment access programs. Today's decision by Shire does not have any impact on the treatment of REPLAGAL patients outside the US.