Shire filed a 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 European Union 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.

Fast Track designation is an FDA-approved process that facilitates the development and expedites the review of drugs to treat serious diseases and fill an unmet medical need with the goal of getting important new treatments to patients earlier. This process allows a company to file the sections of the BLA as they become available instead of filing all the sections at once. It also enables the agency to commence its review and proceed on a rolling basis as the additional sections are completed and submitted for review.

“We will continue to work closely with the FDA in the coming months on the rolling BLA submission for REPLAGAL, “said Sylvie Grégoire, president, Shire Human Genetic Therapies. “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 U.S. 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 U.S.

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