Shire filed a Biologics License Application (BLA) for Replagal in December 2009. The FDA requested additional human pharmacokinetic data to confirm comparability between product that was manufactured in roller bottles, and that which is manufactured in bioreactors. Product made by the bioreactor process is already approved for commercial use in the EU as well as a number of other countries.
As a result of this request, Shire withdrew its December BLA filing, and, at the suggestion of the FDA, requested and received Fast Track designation. Shire will immediately initiate the rolling submission of the Replagal BLA, and will submit the requested pharmacokinetic data around mid-year.
Sylvie Gregoire, president of Shire Human Genetic Therapies, said: “We will continue to work closely with the FDA in the coming months on the rolling BLA submission for Replagal. We remain committed to continuing to provide Fabry patients in the United States with Replagal under the treatment protocol.”
Replagal is currently approved for the treatment of fabry disease in 45 countries and has been available to US patients since December 2009 under an FDA-approved treatment protocol filed at the request of FDA. The Replagal early access program was put in place as a result of the supply disruption of the only currently marketed treatment for Fabry disease in the US.