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and so­cial care com­mit­tee chair Sarah Wol­las­ton asked Ver­tex ex­ec­u­tives how much Orkam­bi stock had gone out of date over the past year.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“I do not be­lieve that we have had any stock go out of date and be de­stroyed…we do not pack­age it up un­til we are due to be sell­ing it,” Ver­tex chief com­mer­cial of­fi­cer Stu­art Ar­buck­le re­spond­ed.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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.