No­var­tis' SMA gene-ther­a­py, if priced at $2M, could be more cost-ef­fec­tive than Bio­gen's Spin­raza — ICER

Bio­gen’s Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and of­ten fa­tal ge­net­ic mus­cu­lar dis­or­der. But the price tag of $750,000 for the first year of ther­a­py (and a low­er price there­after) prompt­ed heavy crit­i­cism, al­though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. How­ev­er, a re­port by the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) on Thurs­day has sug­gest­ed No­var­tis’ ex­per­i­men­tal SMA gene ther­a­py, Zol­gens­ma, could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion on the drug in May 2019. In its re­view, ICER has as­sumed Zol­gens­ma will car­ry a list price of $2 mil­lion, al­though the Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the cu­ra­tive ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

De­spite the lack of long-term da­ta on ei­ther ther­a­py, the non-prof­it es­ti­mat­ed in­cre­men­tal cost-ef­fec­tive­ness of Spin­raza is $728,000 per QALY in presymp­to­matic SMA pa­tients, while Zol­gens­ma has an in­cre­men­tal cost-ef­fec­tive­ness of $247,000 per QALY in pa­tients with symp­to­matic Type I SMA (on the ba­sis of a place­hold­er price of $2 mil­lion).

QALYs, or qual­i­ty-ad­just­ed life-years, are a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health.

“No­var­tis has es­ti­mat­ed that Zol­gens­ma would yield 13.3 QALYs in SMA Type I pa­tients, and could be priced to $4-5 mil­lion based on the re­la­tion­ship of 10-year cost of ap­proved drugs for ul­tra-rare dis­eases and their cor­re­spond­ing in­cre­men­tal QALYs gained. ICER es­ti­mates Zol­gens­ma pro­vides 11.33 QALYs, and us­es a place­hold­er price of $2 mil­lion. While ICER un­der­cut both of No­var­tis’ es­ti­mates, the high cost/QALY of Spin­raza in Type I SMA pa­tients of­fers No­var­tis bar­gain­ing pow­er with pay­ers if the com­pa­ny can ar­gue Spin­raza use could be re­duced or elim­i­nat­ed to off­set to­tal costs,” Leerink an­a­lysts wrote in a note on Fri­day.

Cur­rent­ly avail­able da­ta on Spin­raza and Zol­gens­ma show pro­longed sur­vival and im­proved mo­tor func­tion com­pared with his­tor­i­cal con­trols or place­bo, but “there re­mains con­sid­er­able un­cer­tain­ty in the gen­er­al­iz­abil­i­ty of the re­sults and in the long-term dura­bil­i­ty and tol­er­a­bil­i­ty of treat­ment. In par­tic­u­lar, for both in­ter­ven­tions, the nar­row el­i­gi­bil­i­ty cri­te­ria of tri­als and the lim­it­ed sam­ple size (es­pe­cial­ly for Zol­gens­ma) rais­es con­cerns about gen­er­al­iz­abil­i­ty of re­sults to the wider pop­u­la­tion of pa­tients with SMA,” the re­port added.

Bio­gen un­der­scored this dif­fer­ence in sam­ple size in its re­sponse to the re­port. “The draft ICER re­port is an in­com­plete rep­re­sen­ta­tion of Spin­raza’s val­ue to pa­tients and health care sys­tems. Fur­ther­more, to com­pare a treat­ment that has helped near­ly 6,000 pa­tients world­wide against an in­ves­ti­ga­tion­al treat­ment on da­ta from 15 pa­tients with an ar­ti­fi­cial­ly se­lect­ed price is mis­lead­ing and wrong,” a spokesper­son told End­points News.

The ICER re­port is not fi­nal and has been opened to the pub­lic for com­ment, which will be in­cor­po­rat­ed in­to an ev­i­dence re­port in Feb­ru­ary 2019. This re­port will be sub­ject to fur­ther de­lib­er­a­tion by one of ICER’s in­de­pen­dent ev­i­dence ap­praisal com­mit­tees in ear­ly March, af­ter which a fi­nal re­port will be re­leased by the end of the month.

“If con­firmed in a fi­nal re­port…these find­ings could of­fer sup­port for No­var­tis in pric­ing and ac­cess ne­go­ti­a­tions for Zol­gens­ma ahead of 2019 ap­proval,” Leerink an­a­lysts added.

In re­sponse to the re­port, No­var­tis said that the find­ings had af­firmed the com­pa­ny’s ini­tial as­sess­ment of the val­ue of their prod­uct. “While we have not yet de­ter­mined the price of our in­ves­ti­ga­tion­al prod­uct…once we de­ter­mine a price, we are com­mit­ted to flex­i­bly part­ner­ing with health­care stake­hold­ers to en­sure ac­cess,” a spokesper­son told End­points News. 

Like NICE in the UK, ICER an­a­lyzes the ev­i­dence on the ef­fec­tive­ness and val­ue of drugs and oth­er med­ical ser­vices in the Unit­ed States, how­ev­er un­like NICE, it is not a gov­ern­ment-af­fil­i­at­ed body.

In Au­gust, NICE re­fused to rec­om­mend the use of Spin­raza in the UK — de­spite Bio­gen dis­count­ing its price — on the ba­sis that the long-term im­pact of the drug was un­clear for pa­tients, some of whom die by the age of 2. With­out that da­ta, the price of the drug couldn’t be jus­ti­fied, they said.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Uğur Şahin, BioNTech CEO (Andreas Arnold/picture-alliance/dpa/AP Images)

BioN­Tech opens new plas­mid DNA man­u­fac­tur­ing fa­cil­i­ty in Ger­many

German mRNA player BioNTech opened the doors to a new manufacturing facility on Thursday, this one just about 75 miles north of its headquarters in Mainz, Germany.

BioNTech announced on Thursday that it has completed the construction of its first plasmid DNA manufacturing facility in Marburg, Germany. The facility will produce materials for mRNA-based vaccines and therapies along with cell therapies.

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.

BeiGene's new website helps direct cancer patients and caregivers to a wide variety of sources for help.

BeiGene re­veals men­tal health and can­cer care gap in study, de­buts dig­i­tal re­sources

One-fourth of cancer patients are living with depression — and another 20% suffer from anxiety. That’s according to new study results from BeiGene, conducted by Cancer Support Community (CSC), about the mental and emotional health of cancer patients.

While the fact that people with cancer are also dealing with depression or anxiety may not be surprising, what is — and was to BeiGene — is that a majority of them aren’t getting support. 60% of respondents said they were not referred to a mental health professional, and even more concerning, two in five who specifically asked for mental health help did not get it. CSC, a nonprofit mental health in cancer advocacy group, surveyed more than 600 US cancer patients.

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