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

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.

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).