Reshma Kewalramani, Vertex CEO (Vertex)

A year out from FDA ap­proval, Ver­tex looks to ex­pand top-sell­ing CF drug to pe­di­atrics

About a year af­ter get­ting the FDA thumbs-up for its block­buster cys­tic fi­bro­sis drug Trikaf­ta, Ver­tex is pick­ing up where it left off.

The Boston-based biotech read out pos­i­tive re­sults from a Phase III study in­volv­ing chil­dren ages 6 to 11, and an­nounced plans to file for a sup­ple­men­tal NDA lat­er this year. Pre­vi­ous­ly, the triplet reg­i­men was on­ly avail­able to pa­tients 12 and up.

Trikaf­ta, Ver­tex’s $VRTX fourth CF drug, is de­signed for those with at least one F508del mu­ta­tion in the CFTR gene. It cov­ers about 90% of CF pa­tients, un­like the com­pa­ny’s Ka­ly­de­co, which on­ly cov­ers 6% of Amer­i­cans with CF, ac­cord­ing to a STAT re­port. With­in weeks of Trikaf­ta’s Oc­to­ber 2019 launch, it be­came Ver­tex’s top-sell­ing drug. Quar­ter­ly US sales hit $420 mil­lion by the end of Jan­u­ary.

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