Are $3M gene ther­a­pies the new nor­mal? Ex­perts say blue­bird will be a test case for a mar­ket ready for prime time

Drug pric­ing ex­perts gen­er­al­ly agree that blue­bird bio’s two re­cent­ly ap­proved gene ther­a­pies and their mul­ti­mil­lion-dol­lar price tags aren’t go­ing to be one-offs as a wave of new cell and gene ther­a­pies makes its way to the mar­ket.

The FDA’s re­cent ap­provals for blue­bird’s $2.8 mil­lion Zyn­te­glo — with ICER sup­port­ing the price and an 80% re­bate if pa­tients don’t achieve trans­fu­sion in­de­pen­dence — and the $3 mil­lion Skysona, ap­proved un­der ac­cel­er­at­ed ap­proval, are like­ly to be the norm for gene ther­a­py prices mov­ing for­ward, par­tic­u­lar­ly if they can re­duce costs else­where in the health care ecosys­tem, ex­perts said.

Daniel Ol­len­dorf

Daniel Ol­len­dorf, di­rec­tor of val­ue mea­sure­ment & glob­al health ini­tia­tives at the Cen­ter for the Eval­u­a­tion of Val­ue and Risk in Health at Tufts Med­ical Cen­ter, told End­points News in a phone in­ter­view that the trend be­hind mul­ti­mil­lion-plus gene ther­a­pies is an ex­ten­sion of what be­gan with No­var­tis’ $2.1 mil­lion spinal mus­cu­lar at­ro­phy gene ther­a­py Zol­gens­ma, which is still priced at about half of the 10-year cur­rent cost of chron­ic SMA ther­a­py, and be­came a block­buster for No­var­tis last year with more than $1.35 bil­lion in an­nu­al sales.

But the ex­pec­ta­tion is that these high list prices will come with risk-shar­ing agree­ments and re­funds if the prod­ucts don’t work so pay­ers don’t have to bear the full brunt of the fi­nan­cial risk, Ol­len­dorf said. And he not­ed that some gene ther­a­pies don’t lend them­selves as well to track­ing mile­stones, but that isn’t the case for ob­serv­ing trans­fu­sion in­de­pen­dence in those re­ceiv­ing Zyn­te­glo.

Still, when Zyn­te­glo first won Eu­ro­pean ap­proval in 2019, the near­ly $2 mil­lion price tag seemed like a sur­mount­able hur­dle, but the ther­a­py has since been with­drawn as the com­pa­ny strug­gled with re­im­burse­ment, and with its fi­nances, even­tu­al­ly lay­ing off al­most a third of its staff in April. An­a­lysts don’t ex­pect ei­ther of the two re­cent­ly ap­proved gene ther­a­pies to bring in peak sales of more than $100 mil­lion an­nu­al­ly.

Mark Trusheim, strate­gic di­rec­tor of NEWDIGS at the Cen­ter for Bio­med­ical Sys­tem De­sign at Tufts Med­ical Cen­ter, told End­points that he ex­pects about 64 prod­uct-in­di­ca­tion ap­provals for cell and gene ther­a­pies through 2030, with about half of those in this durable gene ther­a­py space that can com­mand these mul­ti­mil­lion-dol­lar list prices.

“Mov­ing for­ward, each new prod­uct will be judged on the spe­cif­ic ben­e­fits its ev­i­dence demon­strates,” Trusheim not­ed. “That said, more such list prices be­fore dis­counts and out­comes con­tracts re­sults would seem like­ly in the fu­ture.”

But the ques­tion of whether such high list prices are sus­tain­able has been on the minds of even the reg­u­la­tors who aren’t al­lowed to fac­tor prices in­to their reg­u­la­to­ry de­ci­sions.

“Re­im­burse­ment is the 800-pound go­ril­la in the room,” CBER di­rec­tor Pe­ter Marks not­ed at an event in May, as he mused that if there was bet­ter man­u­fac­tur­ing of small­er batch­es of AAV vec­tors for gene ther­a­pies, for ex­am­ple, he said, there might be a way to put to­geth­er a port­fo­lio of these small­er-pop­u­la­tion prod­ucts that would then be vi­able.

Mov­ing for­ward, com­pa­nies may look to take their gene ther­a­pies to the US first as a test for wider ac­cep­tance, and as the price points are typ­i­cal­ly high­er here than in Eu­rope, Ol­len­dorf said.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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User fees in ac­tion: FDA un­veils new short­ened sup­ple­ment re­view, rare dis­ease pi­lots

Thanks to PDUFA VII, signed into law last Friday by President Joe Biden, the FDA this week unveiled two new industry-friendly pilot programs to advance new rare disease endpoints via additional meetings, and to shorten FDA review times for supplemental apps aimed at unmet medical needs.

The agency this week released eagerly-awaited details behind the shortened pilot, known as the Split Real Time Application Review or STAR pilot program, which will speed up certain FDA reviews of efficacy supplements across all therapeutic areas (thanks to earlier submissions of data), but only for those that propose addressing an unmet medical need.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Al Sandrock, Voyager Therapeutics CEO

Af­ter 12 months of dig­ging, Pfiz­er opts for one of two Voy­ager cap­sids for gene ther­a­py

The path at Voyager Therapeutics keeps winding, and at the 12-month deadline, Pfizer has elected to only move forward with one of two capsids out of the preclinical biotech for the Big Pharma’s AAV gene therapies.

Last October, the Big Pharma and biotech linked arms on a deal that gave Pfizer the ability to hit the gas pedal on two capsids: one for a cardiac target and the other for a rare neurologic disease target.