Mer­ck dis­card jumps straight to the top of the chart of the most ex­pen­sive drugs in the US at $1M-plus

Back late last fall, when Eiger Phar­ma­ceu­ti­cals $EIGR com­plet­ed its vic­to­ry lap with an old Mer­ck dis­card it had de­vel­oped for rare cas­es of prog­e­ria, the com­pa­ny told re­porters that it was think­ing of pric­ing the new­ly dubbed Zok­in­vy at the usu­al rate re­served for ul­tra rare dis­eases. Mer­ck aban­doned the drug years ago af­ter giv­ing up on a can­cer in­di­ca­tion, and let Eiger have it for Prog­e­ria sev­er­al years ago with­out tack­ing on any mile­stones in the deal.

It turns out, the small cap play­er may have gone one or two steps past the usu­al point, though.

GoodRx just came out with their new top 10 list of the most ex­pen­sive drugs in the US. And Zok­in­vy makes the list as the new num­ber 2 ther­a­py with an av­er­age an­nu­al cost of $1,032,480.

Right be­hind it is the sec­ond new en­try on the list: Danyelza, the new drug from Y-mAbs Ther­a­peu­tics that weighs in just un­der 7 fig­ures, at $977,664. That drug is ap­proved as a com­bi­na­tion treat­ment for high-risk cas­es of neu­rob­las­toma. A group of ex-Gen­mab ex­ecs li­censed this in when they grabbed a pair of drugs from Memo­r­i­al Sloan Ket­ter­ing.

No­var­tis holds on to the num­ber 1 spot, way out front with their SMA ther­a­py Zol­gens­ma, just now be­ing cleared in the UK at a dis­count price. In the US, the drug sells for $2.1 mil­lion, and can be cu­ra­tive if the dis­ease is caught ear­ly enough.

Phar­ma de­fend­ers are quick to point out that the cost of the drug still qual­i­fies as a big saver com­pared to the cost of tend­ing to these in­fant pa­tients suf­fer­ing from a ter­ri­ble and lethal dis­ease. Pay­ers who have a hard time cov­er­ing the cost, notes GoodRx, can pay over­time in 5 an­nu­al in­stall­ments of $425,000.

What­ev­er the ar­gu­ments, though, price tags like these tend to at­tract con­sid­er­able out­rage among the peo­ple who face out-of-pock­et drug costs that can rou­tine­ly force them off their med­ica­tion sched­ule. And that makes the top 10 price list of in­ter­est to every­one in the in­dus­try, par­tic­u­lar­ly as small out­fits like Eiger and Y-mAbs join the ma­jor play­ers on the pricey ros­ter.

Of the oth­er 7 drugs on the list, 4 have hiked the price.

  • Myalept from Aege­ri­on jumped from $71,306 to $74,159 per month — an an­nu­al cost weigh­ing in at $889,904. The ther­a­py is used to treat lep­tin de­fi­cien­cy.
  • Brineu­ra, which treats one form of Bat­ten dis­ease, rose 2.14%, re­ports GoodRx, to $730,340 a year. That breaks down to 300 mg every 2 weeks.
  • The price of Am­gen’s Blin­cy­to, num­ber 8 on the list, was hiked by 5.89% to $712,672.
  • Hori­zon Ther­a­peu­tics, which rou­tine­ly makes an ap­pear­ance in the ul­tra-ex­pen­sive list, in­creased the price of Rav­ic­ti 4.8% to $695,970. That’s num­ber 9.

At num­ber 10 we have Alex­ion’s Soliris — long the one-time reign­ing champ in the most-ex­pen­sive-drugs cat­e­go­ry. It now sells for $678,392 as Alex­ion makes way for a suc­ces­sor ther­a­py — and As­traZeneca looks to take over the fran­chise once it com­pletes a $39 bil­lion ac­qui­si­tion.

That leaves Lux­tur­na, a break­through eye gene ther­a­py ac­quired by Roche which sells for $850,000 for a pair of eyes — or $425,000 each. Folo­tyn for a rare blood can­cer costs $793,870.

Here’s the straight list from GoodRx, ranked 1 through 10.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

Covid-19 roundup: J&J boost­er shot da­ta show promise; CD­C's ACIP meet­ing this week to dis­cuss Pfiz­er boost­ers

J&J revealed a summary of new Covid-19 vaccine data today, including new results showing booster shots may help with protection.

A Phase III study (ENSEMBLE 2) looked at booster shots at two different points in time: a second shot 56 days after the first shot, or a second shot six months after the first. The eight-week shot showed increased protection against symptomatic Covid-19, with the following levels of protection: