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.


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

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In a late-night tweet Wednesday, the Tesla chief announced:

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George Yancopoulos (Regeneron)

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

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Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi preps an­oth­er round of lay­offs as new ex­ecs look to slim down the glob­al phar­ma in an on­go­ing re­or­ga­ni­za­tion — re­port

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