Keywords: Shire, USA, Replagal, Velaglucerase, Fabry disease, Gaucher disease, BLA filing

Shire submits Replagal BLA with the USA’s FDA; recaps velaglucerase status

Article | 23 December 2009

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Specialty biopharmaceutical company Shire says that it has submitted a Biologics License Application (BLA) with the USA’s Food and Drug Administration for Replagal (agalsidase alfa), its enzyme replacement therapy for Fabry disease. The drug first received marketing authorization in the European Union in 2001, and is approved for the treatment of Fabry disease in 45 countries.

The news saw Shire’s share price edge up 0.6% to £11.98, close to its two-year high, having dropped to under £8 at the start of March this year. 

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Replagal is currently available to US Fabry patients under an FDA-approved treatment protocol, and the company is also supporting emergency Investigational New Drug 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 USA, as a result of problems at US biotechnology major Genzyme, which has been hit by trouble at a manufacturing site in Boston, where contamination of a bioreactor with a virus has hampered production (The Pharma Letter November 27).

"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," she noted. Shire expects its Replagal supply to be adequate to meet anticipated global demand.

Update on Shire's potential Gaucher drug

In light of the supply restrictions on one of the commercially-available products for Gaucher disease (notably Genzyme’s Cerezyme; imiglucerase for injection) 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 USA, European Union and Canada. In the USA, 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 MA.

• 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 US New Drug Application for velaglucerase alfa.

• Shire continues to work with US physicians to provide access to velaglucerase alfa under an FDA-approved treatment protocol. In Europe and other countries outside the USA, patients continue to receive the product through pre-approval access programs.