Pub­lic Cit­i­zen picks apart Cures 2.0 draft leg­is­la­tion, crit­i­ciz­ing sev­er­al RWE pro­vi­sions

Con­sumer ad­vo­ca­cy or­ga­ni­za­tion Pub­lic Cit­i­zen on Thurs­day sent a let­ter to con­gres­sion­al lead­ers, seek­ing changes to a key piece of leg­is­la­tion that will cre­ate new FDA re­forms if it’s en­act­ed.

The or­ga­ni­za­tion took par­tic­u­lar is­sue with the bill, which is the sec­ond it­er­a­tion of the mam­moth 21st Cen­tu­ry Cures Act or Cures 2.0, as it would in­crease the FDA’s re­liance on re­al-world ev­i­dence in cer­tain cir­cum­stances, such as ver­i­fy­ing the clin­i­cal ben­e­fit of drugs fol­low­ing ac­cel­er­at­ed ap­provals. Pub­lic Cit­i­zen said that would:

sub­stan­tial­ly weak­en the FDA’s post-ap­proval study re­quire­ments for new drugs ap­proved un­der the ac­cel­er­at­ed-ap­proval path­way by al­low­ing the FDA to re­ly on analy­ses of clin­i­cal care da­ta repos­i­to­ries, pa­tient reg­istries, and oth­er sources of re­al world ev­i­dence — rather than well-de­signed, ran­dom­ized, con­trolled clin­i­cal tri­als — to con­firm that drugs ap­proved un­der this reg­u­la­to­ry path­way based on their ef­fect on a sur­ro­gate end­point have an ef­fect on ir­re­versible mor­bid­i­ty or mor­tal­i­ty or oth­er clin­i­cal ben­e­fit.

On the sec­tion of Cures 2.0 that would pro­vide $11 bil­lion to es­tab­lish a new sub­scrip­tion con­tract pro­gram for cer­tain an­tibi­otics, Pub­lic Cit­i­zen writes in its let­ter to House Speak­er Nan­cy Pelosi (D-CA) and mi­nor­i­ty leader Kevin Mc­Carthy (R-CA) that the pro­gram “would be un­like­ly to spur de­vel­op­ment of tru­ly in­no­v­a­tive drugs to com­bat in­fec­tions re­sis­tant to cur­rent treat­ments…A sub­scrip­tion mod­el is not suit­able for crit­i­cal-need an­tibi­otics be­cause un­like he­pati­tis C treat­ments, there is not ur­gent un­met need dri­ven by high prices do­mes­ti­cal­ly, and there may not be com­pet­ing ther­a­pies that are clin­i­cal­ly sub­sti­tutable.”

An­na Es­hoo

House law­mak­ers have al­ready sug­gest­ed com­bin­ing the Cures 2.0 bill with a new mul­ti-bil­lion-dol­lar NIH cen­ter, to be known as ARPA-H, which would seek to con­duct more high-risk, high-re­ward re­search along the lines of DARPA.

“ARPA-H and Cures 2.0 are com­ple­men­tary and the chair will seek to move them to­geth­er so that we can ad­vance the leg­is­la­tion, not on­ly through the full com­mit­tee, but through the full House of Rep­re­sen­ta­tives,” Cal­i­for­nia’s An­na Es­hoo, who serves as chair­woman of the House En­er­gy & Com­merce sub­com­mit­tee on health, said in a state­ment ear­li­er this month.

Pub­lic Cit­i­zen al­so seeks to add sev­er­al pro­vi­sions to the Cures re­dux bill, in­clud­ing a rule to al­low gener­ic drug man­u­fac­tur­ers to up­date their la­bels more prompt­ly to re­flect cer­tain types of new­ly ac­quired in­for­ma­tion re­lat­ed to drug safe­ty, re­gard­less of whether the re­vised la­bel­ing dif­fers from the brand name drug (the FDA cur­rent­ly re­quires gener­ic and brand la­bels to be iden­ti­cal), adding:

New safe­ty is­sues com­mon­ly arise af­ter gener­ic ver­sions have en­tered the mar­ket, un­der­scor­ing the im­per­a­tive of main­tain­ing in­cen­tives for ro­bust man­u­fac­tur­er sur­veil­lance of safe­ty con­cerns through­out the life of a drug prod­uct. The Food and Drug Ad­min­is­tra­tion (FDA) cur­rent­ly does not al­low gener­ic drug man­u­fac­tur­ers to ini­ti­ate safe­ty up­dates to prod­uct la­bel­ing when they be­come aware of new risks, al­though brand-name man­u­fac­tur­ers have long had that abil­i­ty and re­spon­si­bil­i­ty. Al­though the FDA in 2013 pro­posed a new rule to cor­rect this safe­ty gap, the rule was not fi­nal­ized and was lat­er with­drawn in 2018.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; 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.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Days af­ter re­port­ing PhI­II fail­ure, GSK pulls BC­MA drug from US mar­ket — but it's not giv­ing up en­tire­ly yet

GSK is pulling its BCMA-targeting drug from the US market, ending a short, two-year run for a high-profile product that, among other things, was hailed for marking the pharma giant’s return to oncology.

The company is initiating the process for withdrawal at the request of the FDA, which in turn was based on the negative readout of a confirmatory Phase III trial earlier this month. In that trial, GSK’s Blenrep failed to extend progression-free survival over standard of care for patients with multiple myeloma who have received at least two prior lines of therapy.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.