Clin­i­cal un­cer­tain­ty, price push­es UK's NICE to spurn Akcea's Waylivra

Echo­ing some of the con­cerns the FDA un­der­scored be­fore hand­ing its re­jec­tion, the UK’s cost-ef­fec­tive­ness watch­dog NICE has al­so for­sak­en Akcea’s drug, volane­sors­en.

On Fri­day, NICE is­sued a draft rec­om­men­da­tion re­ject­ing the fa­mil­ial chy­lomi­cron­aemia syn­drome (FCS) drug, cit­ing a myr­i­ad of rea­sons in­clud­ing un­cer­tain­ty un­der­pin­ning its clin­i­cal ev­i­dence as well as price. The drug is en­gi­neered to work by di­min­ish­ing the pro­duc­tion of ApoC-III, a pro­tein that reg­u­lates plas­ma triglyc­erides. FCS is caused by in­suf­fi­cient or im­paired func­tion of an en­zyme re­spon­si­ble for break­ing down triglyc­erides. It af­fects be­tween 55 and 110 peo­ple in Eng­land.

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