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.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

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Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.

Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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