CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Cen­ters for Medicare and Med­ic­aid Ser­vices on Tues­day said it will on­ly pay for Bio­gen’s Aduhelm and oth­er FDA-ap­proved an­ti-amy­loid mon­o­clon­al an­ti­bod­ies for Alzheimer’s dis­ease un­der CMS-ap­proved ran­dom­ized con­trolled tri­als.

The draft na­tion­al cov­er­age de­ci­sion, which in­sur­ers na­tion­wide are like­ly to fol­low, makes clear that CMS will be look­ing for ran­dom­ized con­trolled tri­als that “demon­strate a clin­i­cal­ly mean­ing­ful ben­e­fit in cog­ni­tion and func­tion.” That will be a tough task for Bio­gen, which pre­vi­ous­ly showed con­flict­ing ben­e­fits from past Aduhelm tri­als that were ini­tial­ly cut short due to fu­til­i­ty and then res­ur­rect­ed for the ac­cel­er­at­ed ap­proval.

“None of the tri­als com­plet­ed as of the pub­li­ca­tion of this NCA has con­vinc­ing­ly demon­strat­ed that use of an­ti­amy­loid mAbs re­sult in a mean­ing­ful im­prove­ment in health out­comes for AD pa­tients,” CMS said in its draft de­ci­sion, which is now open for 30 days of more com­ments, and will be fi­nal­ized in April.

Hol­ly Fer­nan­dez Lynch, a bioethics pro­fes­sor at the Uni­ver­si­ty of Penn­syl­va­nia, told End­points News that she thinks CMS made the right de­ci­sion in go­ing for cov­er­age with ev­i­dence de­vel­op­ment (CED) and re­quir­ing the tri­al, al­though she not­ed how odd it is that the gov­ern­ment will be es­sen­tial­ly pay­ing Bio­gen to run a tri­al.

“I didn’t think out­right re­fusal of any cov­er­age was gen­uine­ly on the ta­ble. This is a pret­ty strong re­buke of FDA as it is,” she said.

The an­nounce­ment is a blow for Bio­gen, which will like­ly have to de­sign and run an­oth­er clin­i­cal tri­al in ad­di­tion to its con­fir­ma­to­ry tri­al to sat­is­fy the FDA’s ac­cel­er­at­ed ap­proval re­quire­ments. Bio­gen’s stock dropped about 8% Tues­day af­ter­noon.

“This draft cov­er­age de­ter­mi­na­tion de­nies the dai­ly bur­den of peo­ple liv­ing with Alzheimer’s dis­ease,” Bio­gen said in a state­ment emailed to End­points, adding:

Cov­er­age with ev­i­dence de­vel­op­ment (CED) un­der a ran­dom­ized clin­i­cal tri­al will ex­clude al­most all pa­tients who may ben­e­fit. This will sig­nif­i­cant­ly lim­it pa­tient ac­cess to an FDA-ap­proved treat­ment, es­pe­cial­ly for un­der­served pa­tients as ev­i­denced in oth­er CED de­ter­mi­na­tions. CEDs can take months to years to ini­ti­ate, and hun­dreds of Alzheimer’s pa­tients – the ma­jor­i­ty of whom are Medicare ben­e­fi­cia­ries – are pro­gress­ing each day from mild to mod­er­ate dis­ease stages, where treat­ment may no longer be an op­tion.

But Lon Schnei­der, who di­rects the Uni­ver­si­ty of South­ern Cal­i­for­nia’s State of Cal­i­for­nia Alzheimer’s Dis­ease Cen­ter, told End­points that 80% of pa­tients in the Bio­gen tri­als had pro­dro­mal AD, which is “de­f­i­n­i­tion­al­ly, vir­tu­al­ly no dai­ly bur­den (a bit of mem­o­ry im­pair­ment, yes, a bit of amy­loid? Yes). And the tri­als couldn’t demon­strate that any one pa­tient ben­e­fit­ted from ad­u­canum­ab, so who’s be­ing de­prived ben­e­fit?”

He called the move “a smack­down” as CMS “al­so told Lil­ly that FDA might give you AA [ac­cel­er­at­ed ap­proval] or BTD [break­through des­ig­na­tion] but we’ll wait to see pos­i­tive tri­als re­sults.”

In­dus­try group PhRMA of­fered a sim­i­lar char­ac­ter­i­za­tion, not­ing, “With this pro­pos­al, CMS is writ­ing off an en­tire class of med­i­cines be­fore mul­ti­ple prod­ucts have even been re­viewed by FDA, po­si­tion­ing it­self and not FDA as the key ar­biter of clin­i­cal ev­i­dence.”

The FDA de­clined to com­ment on the de­ci­sion by CMS.

CMS al­so lays out very spe­cif­ic tri­al re­quire­ments that Bio­gen, Roche, Eli Lil­ly and any oth­er amy­loid con­tenders will have to meet, which is not what CMS has done in pre­vi­ous na­tion­al cov­er­age de­ci­sions, not­ing that it’s aware of at least three oth­er an­ti-amy­loid mAbs cur­rent­ly ap­proach­ing Phase 3 tri­als.

“The di­ver­si­ty of pa­tients in­clud­ed in each tri­al must be rep­re­sen­ta­tive of the na­tion­al pop­u­la­tion di­ag­nosed with AD,” CMS says, not­ing that the tri­als must ad­dress, “at a min­i­mum,” whether the  use of mAbs di­rect­ed against amy­loid re­sult in a sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­ing­ful dif­fer­ence in de­cline in cog­ni­tion and func­tion, and whether they ad­dress the ad­verse events as­so­ci­at­ed with the use of these an­ti-amy­loid mAbs.

The news comes as Bio­gen re­cent­ly cut Aduhelm’s price in half, af­ter sell­ing just $300,000 of Aduhelm in the ear­ly months, and com­ing un­der heavy crit­i­cism for the $56,000 price tag that forced CMS to prep for record-high pre­mi­um in­creas­es for se­niors. HHS Sec­re­tary Xavier Be­cer­ra ear­li­er this week called on CMS to re­con­sid­er those steep pre­mi­um in­creas­es now that Aduhelm’s price has been re­duced.

Sen. Bernie Sanders of Ver­mont said in a state­ment that CMS’ de­ci­sion “is an im­por­tant step for­ward, es­pe­cial­ly fol­low­ing Sec­re­tary Be­cer­ra’s move yes­ter­day to di­rect Medicare to re­con­sid­er a pre­mi­um rate hike caused in part by the out­ra­geous price of Aduhelm.”

Oth­ers who be­lieve that Aduhelm does work were dis­ap­point­ed by the de­ci­sion from CMS, which will like­ly mean on­ly a lim­it­ed num­ber of pa­tients are cov­ered un­der a CMS-ap­proved clin­i­cal tri­al.

“This is clear­ly dis­ap­point­ing news, as CED would sig­nif­i­cant­ly lim­it the num­ber of pa­tients who can ac­cess Aduhelm and oth­er an­ti-Abe­ta mAbs, and in this sce­nario, we would ex­pect a pro­longed process to ini­ti­ate and en­roll pa­tients in­to the CED clin­i­cal tri­als which need to be re­viewed and ap­proved by CMS,” SVB Leerink told in­vestors.

Ad­vo­ca­cy or­ga­ni­za­tions were sim­i­lar­ly not pleased.

Har­ry Johns, Alzheimer’s As­so­ci­a­tion CEO, said in a state­ment, “To­day’s draft de­ci­sion by the Cen­ters for Medicare & Med­ic­aid Ser­vices (CMS) is shock­ing dis­crim­i­na­tion against every­one with Alzheimer’s dis­ease, es­pe­cial­ly those who are al­ready dis­pro­por­tion­ate­ly im­pact­ed by this fa­tal dis­ease, in­clud­ing women, Blacks and His­pan­ics.”

George Vraden­burg, chair and co-founder of Us­Again­stAlzheimer’s, added, “This is ab­solute­ly un­ac­cept­able. If this de­ci­sion stands, for the first time in his­to­ry, mil­lions of Amer­i­cans will be de­nied cov­er­age not just to a drug, but to a whole class of drugs—not by the agency that reg­u­lates drugs but by the fed­er­al in­sur­ance bu­reau­cra­cy.”

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with ad­di­tion­al com­ments.

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

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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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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Giovanni Caforio, Bristol Myers Squibb CEO (Nicolas Messyasz/Sipa via AP Images)

Bris­tol My­ers turns at­ten­tion to new prod­ucts in wake of Revlim­id patent loss

Bristol Myers Squibb CEO Giovanni Caforio is shifting his focus to newer products as generic sales continue to gnaw at the company’s blockbuster myeloma drug Revlimid.

Both Revlimid and Abraxane sales took a dive last year thanks to generic rivals, BMS reported in its Q4 and full-year results on Thursday. As a result, Q4 sales dipped 5% and full-year sales remained flat. However, Caforio sees a silver lining — or rather, two of them.

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Rob Davis, Merck CEO

Mer­ck’s Keytru­da nears $21B in sales, dou­bles down on com­bo tri­als

Merck’s cancer immunotherapy Keytruda notched sales of $20.9 billion in 2022, cementing its status as one of the world’s top-selling drugs. However, it’s far from resting on that accomplishment.

Merck executives touted nine ongoing trials in its annual earnings call on Thursday, including five studies in Phase III, for Keytruda (pembrolizumab) in combination with other immuno-oncology drugs. The trials include combinations with Merck’s own developments as well as other pharma companies’ candidates, including its melanoma collaboration with Moderna and its mRNA technology plus Keytruda, aimed at creating a personalized vaccine treatment to reduce the risk of cancer recurrence or death.

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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.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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Lars Fruergaard Jørgensen, Novo Nordisk CEO (Christopher Goodney/Bloomberg via Getty Images)

No­vo Nordisk notch­es big GLP-1 sales amid re­bound­ing sup­plies, but cau­tions on fu­ture 'pe­ri­od­ic con­straints'

With Novo Nordisk’s obesity treatment Wegovy fully back in stock in December, sales are beginning to soar, the Danish pharma reported during its annual earnings call on Wednesday. Total scripts of the glucagon-like peptide 1 (GLP-1) Wegovy topped 37,000 weekly in mid-January, a hockey stick uptick from end-of-year levels below 15,000 per week.

The new prescriptions come on top of the overall momentum of Novo obesity drug sales in 2022, although the then supply-constrained Wegovy was only part of that. Sibling obesity med Saxenda accounted for DKK 10.7 billion ($1.58 billion) of the total DKK 16.9 billion ($2.49 billion), or about 63%, in Novo Nordisk’s reported obesity segment sales.

Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)

Go­ing block­buster hunt­ing, Vas Narasimhan out­lines vi­sion for ‘pure-play’ No­var­tis

By the time 2023 is over, Novartis expects to be moving much lighter: It will have spun out its generics subsidiary Sandoz, completed layoffs of thousands of staffers worldwide and put in new internal structures for running the company.

And it will be ready to hunt for blockbusters.

CEO Vas Narasimhan underscored Novartis’ upcoming transformation into a “pure-play” company, reiterating across a series of calls — one with reporters, two with investors and analysts — that the new structure would boost its R&D productivity and sharpen its focus on big, new medicines.

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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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