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withdrawn its BLA for Replagal agalsidase alfa to treat Fabry's disease less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.
Approval of Shire's BLA would
have ensured U.S. patients access to the drug, and a choice of treatments when
Fabrazyme supplies come back on line. Patients in the U.S. will not have this
option because Shire decided to withdraw the BLA after receiving the advisory
committee briefing documents, which the company has said indicated FDA would
require additional controlled clinical trial data.
Shire has not disclosed what in
the documents specifically led it to reach that conclusion.
But after the BLA was pulled,
BioCentury received from an anonymous source an unsolicited package of
documents that are labeled as FDA briefing materials prepared for the March 27,
2012, meeting of the Cardiovascular and Renal Drugs Advisory Committee.