FDA ex­pands ap­proval for Ver­tex's CF drug Orkam­bi to in­fants un­der two years of age

The FDA on Fri­day ex­pand­ed its ap­proval of Ver­tex’s block­buster cys­tic fi­bro­sis pill Orkam­bi to in­clude chil­dren be­tween 12 and 24 months.

Orkam­bi first won ap­proval in 2015 for ages 2 years and old­er with two copies of the F508del mu­ta­tion, but Ver­tex went for the ear­li­er age in­di­ca­tion, as CMO Car­men Boz­ic said in a state­ment: “Treat­ing chil­dren with cys­tic fi­bro­sis as ear­ly in life as pos­si­ble is crit­i­cal­ly im­por­tant, be­cause ear­ly treat­ment has the po­ten­tial to slow the pro­gres­sion of this dev­as­tat­ing dis­ease.”

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