Shire plc, the global specialty biopharmaceutical company, announced plans to file a Biologics License Application (BLA) with the US Food and Drug Administration (FDA) for Replagal (agalsidase alfa), its enzyme replacement therapy for Fabry disease, by the end of the year. The company also announces that a treatment protocol for Replagal, filed at the request of FDA, has been approved, and that it will support emergency IND requests. The early access program is being put in place in view of the announced supply restriction of the only currently marketed treatment for Fabry disease in the United States.

“We are eager to have the opportunity to offer Replagal to Fabry patients in the United States,” commented Sylvie Grégoire, president of Shire Human Genetic Therapies. “There is an abundance of long-term experience with Replagal in Fabry patients since the product has been commercially available outside of the United States since 2001.”

The treatment protocol and emergency IND will allow physicians to treat Fabry patients with Replagal in advance of potential commercial availability in the US Shire will initially provide Replagal free of charge to patients. The current supply of Replagal is projected to be adequate to meet anticipated global demand.

Replagal is a human form of enzyme alpha-galactosidase A (a-Gal A) manufactured in a human cell line by gene activation. REPLAGAL is approved in 45 countries worldwide.