FDA ap­proves Am­i­cus' Pom­bil­i­ti + Op­fol­da com­bo ther­a­py for Pompe dis­ease

The FDA on Thurs­day ap­proved Am­i­cus Ther­a­peu­tics’ com­bo ther­a­py for a rare in­her­it­ed lyso­so­mal dis­or­der called Pompe dis­ease fol­low­ing sev­er­al reg­u­la­to­ry de­lays.

The drugs are mar­ket­ed as Pom­bil­i­ti (cipaglu­cosi­dase al­fa) and Op­fol­da (miglu­s­tat). Pom­bil­i­ti is a long-term en­zyme re­place­ment ther­a­py and Op­fol­da is an en­zyme sta­bi­liz­er. The ther­a­py is al­ready ap­proved in the EU, and the UK’s Na­tion­al In­sti­tute for Health and Care Ex­cel­lence rec­om­mend­ed it last month as a first-line ther­a­py as well as in lat­er lines of treat­ment af­ter it re­ceived ap­proval there.

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