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.

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Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

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Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

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Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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