Date: 2012-03-15
Type of information: Withdrawal of a market application in the US
Product name: Replagal®
Compound: agalsidase alfa
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
Company: Shire (UK - USA)
Disease: Fabry disease
Latest news: * On March 14, 2012, Shire has announced that it has withdrawn its Biologics License Application (BLA) for Replagal® (agalsidase alfa) with the FDA. Shire has been in ongoing dialogue with the FDA since the supply shortage of the only US approved treatment for Fabry disease. In 2009, and again in 2011, the FDA encouraged Shire to submit an application for the approval of Replagal®. The information in the application included relevant updates such as manufacturing and open long-term clinical trial data. These discussions led the Company to file a BLA last November in anticipation of a quick review process and eventual approval - allowing Shire to supply more US patients with a therapy they desperately needed at the time. Recent interactions with the FDA have led the Company to believe that the agency will require additional controlled trials for approval. No concerns over the product’s safety profile were raised by the FDA. Shire has concluded that the likely additional studies would cause a significant delay, and an approval of Replagal® for US patients would only be possible in the distant future. Shire has therefore decided to withdraw its BLA. Over 1000 patients have switched to Replagal® since the beginning of the supply shortage in mid-2009. Shire has been providing Replagal® free of charge to around 140 US patients – about 20% of the treated US patients - through treatment access programs.
Patents:
Submission of marketing authorization application USA : 2011-11
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA: 2012-03-14
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization: 2001-08-03
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 1998-06-22
Orphan status UE: 2000-08-08
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
