Shire has filed its Fabry disease treatment Replagal for approval in the USA amid a shortage of the currently marketed treatment for the disorder, Genzyme Corp’s Fabrazyme.

Replagal (agalsidase alfa) has been on the market in Europe since 2001 and is approved in some 45 countries. It has also been available under an early access programme in the USA, backed by the Food And Drug Administration, since last summer following the temporary shutdown of a Genzyme manufacturing plant which caused the shortage of Fabrazyme (agalsidase beta).

Sylvie Gregoire, president of Shire Human Genetic Therapies, said “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.” The company expects its Replagal supply to be “adequate to meet anticipated global demand”.

Genzyme’s manufacturing problems also led to a shortage of Cerezyme (imiglucerase) for Gaucher disease and Shire’s bid to fill the void is making progress. The UK firm said that the FDA recently completed pre-approval inspections of its Cambridge and Lexington Massachusetts facilities for the manufacturing and testing of its investigational Gaucher drug velaglucerase alfa, “an important milestone” in the review of the drug.

Velaglucerase also benefits from being in pre-approval programmes and the FDA is scheduled to complete its review of the drug by February 28.