Ver­tex’s fight club rule: Block new treat­ments to cys­tic fi­bro­sis pa­tients un­til Orkam­bi is re­im­bursed prop­er­ly

The bare-knuck­le brawl be­tween Ver­tex’s ex­ec­u­tive crew and NICE has now ex­tend­ed to its new cys­tic fi­bro­sis com­bo Symke­vi — match­ing teza­caftor and iva­caftor. 

Late last week the British drug price reg­u­la­tor put out word that it was halt­ing its ap­praisal of Symke­vi — sold in the US as Symdeko — be­cause Ver­tex “has not pro­vid­ed an ev­i­dence sub­mis­sion for this ap­praisal.”

A meet­ing planned for No­vem­ber 8, as a re­sult, has been can­celled.

Ver­tex $VRTX, for its part, turned around to slap NICE , say­ing its “sin­gle tech­nol­o­gy ap­praisal has not kept pace with changes in med­i­cine and has sig­nif­i­cant lim­i­ta­tions in how it cap­tures and val­ues the full ben­e­fits of pre­ci­sion med­i­cines such as teza­caftor/iva­caftor.”

Ver­tex says it will hap­pi­ly re-en­gage “in the con­text of an ap­pro­pri­ate ap­praisal process, which takes in­to ac­count the struc­ture of an in­no­v­a­tive frame­work con­tract for all of Ver­tex’s cys­tic fi­bro­sis med­i­cines, on which we are cur­rent­ly ne­go­ti­at­ing with NHS Eng­land.”

While Ver­tex has been scor­ing steadi­ly ris­ing rev­enue from its mar­ket-lead­ing cys­tic fi­bro­sis drug Orkam­bi in the US, where man­u­fac­tur­ers have con­sid­er­ably more pow­er in uni­lat­er­al­ly set­ting prices, the big biotech has been duk­ing it out with a slate of sin­gle Eu­ro­pean pay­ers not anx­ious to cov­er a drug they con­sid­er far too weak for the price the com­pa­ny wants.

That’s led to some in­ter­est­ing don­ny­brooks. 

In France, the biotech told pa­tients groups they wouldn’t be run­ning clin­i­cal tri­als of their new com­bos be­cause the coun­try’s reg­u­la­to­ry ob­sta­cles made the process “un­pre­dictable” and con­duct­ing the tri­als “would raise false hope.”And in the UK CEO Jeff Lei­den fol­lowed up by ac­cus­ing British Prime Min­is­ter There­sa May and her gov­ern­ment of putting “a low­er val­ue on the life of a CF pa­tient than oth­er coun­tries around the world.”

The back-and-forth has in­clud­ed calls on Ver­tex to re­veal the price it’s de­mand­ing, which so far hasn’t hap­pened. In an email mes­sage Mon­day morn­ing, a Ver­tex spokesper­son not­ed:

Our CEO reached out to both the head of the NHS, Si­mon Stevens and the Sec­re­tary of State for Health and So­cial Care, Matt Han­cock, weeks ago to meet and con­tin­ue dis­cus­sions to find a so­lu­tion that would se­cure ac­cess to our cur­rent and fu­ture med­i­cines for all CF pa­tients in Eng­land, but we have heard noth­ing since that out­reach.

For now, it seems, talk­ing is over. If British pa­tients want their drugs, says Ver­tex, NICE is go­ing to have to bend first. Pa­tients, how­ev­er, will re­main caught be­tween two op­pos­ing forces.

Im­age: Ver­tex HQ.Ver­tex

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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Paul Hudson, Sanofi CEO (via Getty)

Sanofi's $20B buy­out of Gen­zyme pays off again with Eu­ro­pean OK for first Nie­mann-Pick drug

Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease.

The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS.

Proac­tive­ly pre­vent­ing short­ages: New FDA guid­ance spells out which drugs re­quire risk man­age­ment plans

As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA on Thursday published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities.

While drug shortages peaked in 2011, the FDA says in its new 18-page draft guidance that the number of new drug shortages “has declined significantly since” that peak, reaching a low in 2015 and 2016, thanks in part to a new law’s enactment, known as FDASIA, which helped the agency better prevent or mitigate drug supply disruptions and shortages, and clarified cGMP requirements.