We de­stroyed near­ly 8,000 packs of ex­pired Orkam­bi stock last year, Ver­tex tells UK MPs

To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed on Wednes­day that last year close to 8,000 packs (each con­tain­ing a 28-day sup­ply) of Orkam­bi were de­stroyed af­ter cross­ing their ex­piry date. The US drug­mak­er has been locked in ne­go­ti­a­tion with NICE, which has re­fused to al­low the drug in to Eng­land’s Na­tion­al Health Ser­vice un­til Ver­tex of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness.

In a stand­off with UK par­lia­ment ear­li­er this month, Ver­tex chief Jeff Lei­den stood his ground, de­spite be­ing chas­tised by a pletho­ra of MPs for Ver­tex’s pric­ing strat­e­gy, busi­ness mod­el and ethics.

“The prob­lem is we’ve been paint­ed as I think not be­ing will­ing to take the of­fer, the 90% dis­count that Eng­land has made to us. I would ac­tu­al­ly say it dif­fer­ent­ly. We can­not take that of­fer. Not that we won’t take it; we can’t,” he said, em­pha­siz­ing Ver­tex’s ‘ex­tra­or­di­nary’ in­vest­ment in CF R&D — and the promise of its cur­rent and fu­ture med­i­cines.

Im­age: Jeff Lei­den tes­ti­fy­ing in the Com­mons.

Last year, Ver­tex $VRTX raked in $1.26 bil­lion in net Orkam­bi rev­enue.

As part of the pro­ceed­ings, the Com­mons health and so­cial care com­mit­tee chair Sarah Wol­las­ton asked Ver­tex ex­ec­u­tives how much Orkam­bi stock had gone out of date over the past year.

Stu­art Ar­buck­le

“I do not be­lieve that we have had any stock go out of date and be de­stroyed…we do not pack­age it up un­til we are due to be sell­ing it,” Ver­tex chief com­mer­cial of­fi­cer Stu­art Ar­buck­le re­spond­ed. “I would be sur­prised if it was very much.”

On Wednes­day, in a let­ter to the com­mit­tee Ver­tex said 7,880 packs of Orkam­bi man­u­fac­tured in 2015 and 2016 to sup­ply 13 coun­tries — in­clud­ing the UK — ex­ceed­ed their best be­fore date and were ac­cord­ing­ly dis­card­ed.

“More than 80,000 packs of Orkam­bi have been dis­trib­uted for com­mer­cial sale in the EU since li­cens­ing in 2015, and more than 11,000 packs of Orkam­bi have been sup­plied free of charge…to ap­prox­i­mate­ly 600 pa­tients in Eng­land,” Ver­tex said in the state­ment.

NICE, along with NHS Eng­land, met with Ver­tex last week and agreed to con­tin­ue dis­cus­sions, a NICE spokesper­son told End­points News.

“It is un­eth­i­cal and un­law­ful to sup­ply ex­pired prod­ucts to pa­tients, and we will not do so ir­re­spec­tive of the ac­cess sit­u­a­tion in a giv­en coun­try. We re­main com­mit­ted to work­ing with NHS Eng­land and NICE to reach an ac­cess agree­ment and are con­fi­dent that we will be able to sup­ply ap­proved prod­uct to all el­i­gi­ble Eng­lish pa­tients with­in a few weeks should the gov­ern­ment de­cide to pro­vide ac­cess to our med­i­cines. In the mean­time, we will con­tin­ue to pro­vide free med­i­cines to the sick­est CF pa­tients in Eng­land,” a Ver­tex spokesper­son said in an emailed state­ment on Thurs­day.

The UK has more than 10,400 cys­tic fi­bro­sis pa­tients – the largest CF pop­u­la­tion out­side the US – and rough­ly half the pa­tients car­ry the ge­net­ic mu­ta­tion that could ben­e­fit from Orkam­bi, ac­cord­ing the Cys­tic Fi­bro­sis Foun­da­tion.

“It’s heart­break­ing that pack­ets of life­sav­ing drugs have been thrown away be­cause they’re out of date – what a des­per­ate waste. Thou­sands of peo­ple have suf­fered while these drugs have been sit­ting on the shelf. It is vi­tal the talks that have start­ed de­liv­er a re­al re­sult now. All par­ties in­volved must reach a deal im­me­di­ate­ly so that we can put an end to any fur­ther un­nec­es­sary death and de­te­ri­o­ra­tion,” a spokesper­son told End­points News.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.