Shire announces submission of biologics license for REPLAGAL

Shire has submitted a biologics license application for REPLEGAL (agalsidase alfa) to the Food and Drug Administration (FDA), it has announced.

An enzyme replacement therapy for Fabry disease, the drug is a human form of enzyme alpha-galactosidase A manufactured in a human cell line by gene activation.

It is the only human-cell-line-derived form of enzyme replacement therapy that is indicated for the long-term treatment of Fabry disease patients.

The company has worked closely with the FDA in the past to establish an early access programme in response to the ongoing shortage of the currently marketed treatment for Fabry disease.

“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,” Sylvie Gregoire, president of Shire human genetic therapies commented.

In November, Shire submitted an application to the European Medicines Agency for velaglucerase alfa – its enzyme replacement therapy for the treatment of Type 1 Gaucher disease.