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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

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.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.