(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
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
- 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
- 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