Monday, June 25, 2012
shocked the rare disease community on March 14 when it announced it had
withdrawn its BLA for Replagal agalsidase alfa to treat Fabry's disease less
than two weeks before a scheduled FDA advisory committee meeting - and that it
had no intention to resubmit a marketing application in the U.S.
This week, Shire is set to close down its treatment IND for
Replagal, which has been providing U.S. Fabry's patients with access to the
drug since October 2009. The move will culminate a cascade of events set in
motion by a regulatory crisis that began with the now infamous manufacturing
debacle at Genzyme
Corp., which led to urgent efforts by FDA to find a substitute to compensate
for the shortage of Genzyme's Fabrazyme agalsidase beta, the only drug approved
in the U.S. for Fabry's.