Amicus scores another approval in the UK for Pompe disease therapy, expects FDA OK in Q3

The Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA) in the UK has grant­ed a mar­ket­ing au­tho­riza­tion for Am­i­cus Ther­a­peu­tics’ com­bo ther­a­py for late-on­set Pompe dis­ease, a rare in­her­it­ed lyso­so­mal dis­or­der, the com­pa­ny an­nounced Tues­day.

The drugs, mar­ket­ed as Pom­bil­i­ti (cipaglu­cosi­dase al­fa) and Op­fol­da (miglu­s­tat), have al­so been rec­om­mend­ed as first-line as well as lat­er lines of ther­a­py for the dis­ease by the Na­tion­al In­sti­tute for Health and Care Ex­cel­lence (NICE). Pom­bil­i­ti is a long-term en­zyme re­place­ment ther­a­py while Op­fol­da is an en­zyme sta­bi­liz­er.

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