Amicus has positive meeting with EMA and FDA to accelerate approval of Fabry monotherapy

19 March 2015

Shares of US biotech firm Amicus Therapeutics (Nasdaq: Fold) rocketed 28.2% to $11.99 in pre-market trading today, after the company revealed it has had positive meetings with regulatory authorities in Europe and the USA to discuss the approval pathways for the oral small molecule pharmacological chaperone migalastat as a precision medicine monotherapy for Fabry patients who have amenable genetic mutations.

If approved, migalastat (proposed trade name Amigal under a discontinued licensing accord with GlaxoSmithKline), would be the first oral therapy for Fabry and would compete in the Fabry disease market with the likes of Fabrazyme (agalsidase beta) from Sanofi (Euronext: SAN) subsidiary Genzyme and Shire’s (LSE: SHP) Replagal (agalsidase alfa), both of which are bi-weekly intravenous infusions. Fabrazyme generated 2014 sales of 460 million euros ($484 million), while Replagal revenues were $500 million last year.

Meeting with European Rapporteurs

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