Ver­tex gets NDA go­ing for CF triple com­bo; Dis­arm woos rare dis­ease ex­pert Alvin Shih as CEO

Ver­tex’s has of­fi­cial­ly set sail with its lat­est cys­tic fi­bro­sis triple com­bo, fil­ing an NDA to the FDA. Armed with a break­through ther­a­py des­ig­na­tion, the reg­i­men com­pris­es iva­caftor and teza­caftor — el­e­ments of the biotech’s oth­er CF drugs Ka­ly­de­co and Symdeko — as well as the nov­el com­pound elex­a­caftor (VX-445). An ap­proval can ar­rive as soon as March 2020 as­sum­ing the pri­or­i­ty re­view re­quest is grant­ed, Jef­feries’ Michael Yee notes, adding that the new drug could con­tribute at least $6 bil­lion in peak sales as it taps in­to a new pop­u­la­tion of pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion.

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