FDA ap­proves 5 new cost­ly drugs well ahead of PDU­FA dates

Since 21 Oc­to­ber, the FDA has been on a tear in ap­prov­ing five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were ex­pect­ed to be ap­proved.

For in­stance, the FDA signed off on Ver­tex Phar­ma­ceu­ti­cals’ Trikaf­ta (elex­a­caftor/iva­caftor/teza­caftor), a new treat­ment for those with the most com­mon cys­tic fi­bro­sis mu­ta­tion, af­ter on­ly three months of re­view and well ahead of its 19 March 2020 user fee ac­tion date.

On 14 No­vem­ber, more than three months ahead of its 27 Feb­ru­ary 2020 ac­tion date, the FDA grant­ed ac­cel­er­at­ed ap­proval to BeiGene’s Brukin­sa (zanubru­ti­nib) for the treat­ment of pa­tients with man­tle cell lym­phoma who have re­ceived at least one pri­or ther­a­py.

One day lat­er, No­var­tis’ Adakveo (crizan­l­izum­ab-tm­ca) won ap­proval for its sick­le cell dis­ease treat­ment two months ahead of its PDU­FA date in mid-Jan­u­ary 2020. And yes­ter­day, the FDA grant­ed an ac­cel­er­at­ed ap­proval to an­oth­er sick­le cell drug, Glob­al Blood Ther­a­peu­tics’ Oxbry­ta (vox­elo­tor), three months ahead of its PDU­FA date.

Al­ny­lam Phar­ma­ceu­ti­cals’ Givlaari (givosir­an), mean­while, had a PDU­FA date of 4 Feb­ru­ary 2020, but won ap­proval on 20 No­vem­ber. But oth­er re­cent ap­provals, like SK Life Sci­ences’ Xco­pri (cenoba­mate tablets) to treat par­tial-on­set seizures in adults, and Sh­iono­gi’s com­pli­cat­ed uri­nary tract in­fec­tion drug Fetro­ja (ce­fide­ro­col), won ap­provals near their PDU­FA dates.

The string of quick ap­provals may pro­vide more am­mu­ni­tion for those who crit­i­cize the agency for mov­ing too quick­ly. An ar­ti­cle in JA­MA In­ter­nal Med­i­cine last sum­mer found that few can­cer drugs ap­proved via the ac­cel­er­at­ed ap­proval path­way im­proved sur­vival in con­fir­ma­to­ry tri­als.

How­ev­er, view­ers of the re­cent Sen­ate com­mit­tee hear­ing con­sid­er­ing a new FDA com­mis­sion­er have seen there are still sen­a­tors who be­lieve the FDA is not mov­ing quick­ly enough with some ap­provals.

As the pro­por­tion of new drugs re­ceiv­ing ex­pe­dit­ed ap­provals in re­cent years has been in­creas­ing, so has the num­ber of ap­provals for rare dis­eases.

Janet Wood­cock FDA

Janet Wood­cock, di­rec­tor of the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search, ex­plained re­cent­ly that the agency is work­ing on its own analy­ses to pro­vide “a more ro­bust re­sponse” to these cri­tiques of its ap­proval stan­dards.

She al­so ex­plained how the high num­ber of ap­provals in re­cent years for rare dis­eases may be in­flu­enc­ing this per­cep­tion of a low­er bar, es­pe­cial­ly as more treat­ments are ap­proved on the ba­sis of a sin­gle-arm study or with an ex­ter­nal con­trol group. In ad­di­tion, she point­ed to the “as­tound­ing­ly” high launch prices for some of these rare dis­ease treat­ments that may al­so be part of the rea­son for the push­back.

In­deed, be­fore dis­counts, Trikaf­ta will cost $311,503 an­nu­al­ly, Brukin­sa will cost $12,935 for a 30-day sup­ply, Adakveo will cost be­tween $7,000 and $9,500 per month ($84,000 to $114,000 per year), Oxbry­ta will cost $125,000 per year and Givlaari will cost $575,000 per year.


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