EMA re­moves neg­a­tive note on la­bel of PTC's Duchenne drug; GSK, Ake­bia win Japan OK for oral ane­mia drugs

→ PTC Ther­a­peu­tics $PTCT won con­di­tion­al ap­proval to mar­ket ataluren for Duchenne MD in Eu­rope way back in 2014 — de­spite the fact that all the da­ta point­ed to a fail­ure. And it’s op­er­at­ed with a la­bel say­ing that ef­fi­ca­cy had nev­er been proven for a group of pa­tients. Now the CHMP rec­om­mend­ed by a ma­jor­i­ty of votes to re­move the state­ment “ef­fi­ca­cy has not been demon­strat­ed in non-am­bu­la­to­ry pa­tients” from the Sm­PC for Translar­na. “We are ex­cit­ed to see that the CHMP adopt­ed the pos­i­tive opin­ion for this la­bel mod­i­fi­ca­tion al­low­ing pa­tients who be­come non-am­bu­la­to­ry to con­tin­ue to use Translar­na,” said CEO Stu­art Peltz.

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