Shire submits REPLAGAL BLA with the FDA

Dec 22 2009

Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced that it has submitted a BLA with the FDA for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease. REPLAGAL first received marketing authorization in the European Union in 2001, and is approved for the treatment of Fabry disease in 45 countries.

REPLAGAL is currently available to U.S. Fabry patients under an FDA-approved treatment protocol, and the Company is also supporting emergency IND requests. Shire worked closely with the FDA to establish an early access program in response to the ongoing shortage of the currently marketed treatment for Fabry disease in the U.S.

"We continue to deliver on our commitment to the Fabry community by filing a BLA to support long-term access to REPLAGAL in the United States," said Sylvie Gregoire, President of Shire Human Genetic Therapies. "We understand that this has been a difficult time for patients and we remain committed to doing all we can to support the Fabry community during the supply shortage and for the long-term."

Shire expects its REPLAGAL supply to be adequate to meet anticipated global demand.

Update on Shire's Potential Gaucher Disease Treatment

In light of the supply restrictions on one of the commercially available products for Gaucher disease during the past six months, Shire has provided the following update on recent key activities regarding global access to velaglucerase alfa, its enzyme replacement therapy in development for Type 1 Gaucher disease:

- The Company has submitted marketing applications for velaglucerase alfa in the U.S., EU and Canada. In the U.S., the application is being reviewed by the FDA under Priority Review with a PDUFA date of February 28, 2010. The CHMP has granted accelerated review for the EU MAA. - The FDA recently completed the pre-approval inspections of Shire's Cambridge and Lexington Massachusetts facilities for the manufacturing and testing of velaglucerase alfa. These inspections were an important milestone in the review and approval process for the U.S. NDA for velaglucerase alfa. - Shire continues to work with U.S. physicians to provide access to velaglucerase alfa under an FDA-approved treatment protocol. In Europe and other countries outside the U.S., patients continue to receive the product through pre-approval access programs.

SOURCE Shire Plc