Shire has submitted a Biologics License Application (BLA) to the FDA for Replagal (agalsidase alfa), its enzyme replacement therapy for Fabry disease. Replagal first received marketing authorisation in the EU in 2001, and is approved for the treatment of Fabry disease in 45 countries.
Shire expects its Replagal supply to be adequate to meet anticipated global demand.
Replagal is currently available to US 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 US.
Sylvie Gregoire, president of human genetic therapies at Shire, said: “We continue to deliver on our commitment to the Fabry community by filing a BLA to support long-term access to Replagal in the US. 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.”
Additionally, the company has submitted marketing applications for Velaglucerase Alfa in the US, EU and Canada. 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.
Reportedly, FDA has 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 US NDA for Velaglucerase Alfa.