Martin Shkreli (AP Images)

The FDA OKs gener­ic Dara­prim, the Mar­tin Shkre­li ther­a­py that trig­gered an un­end­ing tem­pest over drug pric­ing

The FDA post­ed a new gener­ic drug ap­proval Fri­day af­ter­noon, and this one ar­rived with a lit­tle ex­tra rel­ish added to the cus­tom­ary pro­nounce­ment.

The agency has giv­en Cerovene the green light to sell gener­ic Dara­prim, Mar­tin Shkre­li’s treat­ment for tox­o­plas­mo­sis that det­o­nat­ed a world class brouha­ha over drug pric­ing that has taint­ed the in­dus­try. Shkre­li no soon­er ac­quired the old, once cheap drug than he in­stant­ly raised the price by more than 5,000%, some­thing that the gen­er­al pub­lic — and a big seg­ment of the bio­phar­ma in­dus­try — was pro­found­ly ap­palled by.

It’s tak­en more than 4 years for a gener­ic to come along, and giv­en the way drug pric­ing works in the U.S., there’s a good chance that re­al price re­lief may still be a long way off.

Shkre­li, al­ways ready to play the Jok­er in every phar­ma biopic, blast­ed back against the pub­lic scorn that he stirred, up un­til he was sen­tenced to 7 years for de­fraud­ing in­vestors in the hedge funds he had dri­ven in­to a brick wall be­fore he turned to biotech — fol­low­ing the path of buy­ing drugs and hik­ing the price.

Law­mak­ers put him in the spot­light, but there was no sham­ing Shkre­li. The price stayed high, and the agency even­tu­al­ly wound up us­ing the case to craft new rules that would help pre­vent Shkre­li wannabes from fol­low­ing in his foot­steps.

“Through the FDA’s Drug Com­pe­ti­tion Ac­tion Plan, we’ve worked to re­move bar­ri­ers in gener­ic drug de­vel­op­ment by not on­ly tak­ing ac­tions that im­prove the ef­fi­cien­cy of the de­vel­op­ment, re­view and ap­proval of gener­ic drugs, but al­so by clos­ing loop­holes that al­low brand-name drug com­pa­nies to ‘game’ the rules in ways that de­lay gener­ic com­pe­ti­tion that Con­gress in­tend­ed,” FDA com­mish Stephen Hahn said in a pre­pared state­ment.

Ac­cord­ing to a re­cent fed­er­al law­suit brought by the FTC and the state of New York, Shkre­li en­gaged in just such be­hav­ior, cook­ing up “an elab­o­rate an­ti­com­pet­i­tive scheme to pre­serve a mo­nop­oly” for Dara­prim. The al­leged of­fens­es ranged from keep­ing sam­ples out of reach for gener­ic drug­mak­ers to block­ing ac­cess to sales rev­enue.

There’s no im­me­di­ate word, though, on what Cerovene plans to charge for the drug. And typ­i­cal­ly, it takes sev­er­al gener­ic drug ri­vals to force the price down sharply. No mat­ter what hap­pens to Dara­prim, though, the drug pric­ing de­bate, and the fall­out that Dara­prim helped cre­ate, has be­come a fix­ture of the US po­lit­i­cal scene and the 2020 elec­tion cy­cle. And some of these can­di­dates are af­ter much big­ger fish than the im­pris­oned Shkre­li.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; 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.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.