FDA clears short­er treat­ment reg­i­men for Ab­b­Vie's hep C treat­ment, Mavyret. Will pa­tients ben­e­fit?

Ab­b­Vie $AB­BV has ob­tained FDA ap­proval to ad­min­is­ter its Mavyret he­pati­tis C pill as a two-month reg­i­men, ver­sus the 12-week course it was orig­i­nal­ly cleared for — a move that may help ex­pand op­tions for the mar­gin of cov­ered pa­tients who need a short­er reg­i­men.

“While over 100,000 pa­tients have been pre­scribed Mavyret for chron­ic HCV in the US, there are still a sig­nif­i­cant num­ber of pa­tients that need op­tions,” Janet Ham­mond, Ab­b­Vie’s vice pres­i­dent of gen­er­al med­i­cine and vi­rol­o­gy ther­a­peu­tic area, said in a state­ment.

Vin­cent Lo Re, a Uni­ver­si­ty of Penn­syl­va­nia School of Med­i­cine pro­fes­sor spe­cial­iz­ing in he­pati­tis, told End­points News that the raft of treat­ments ap­proved since 2014 have been quite ef­fec­tive in cur­ing the dis­ease in most pa­tients. Those who con­tin­ue to suf­fer do so for a va­ri­ety of rea­sons: al­co­holism, drug abuse, men­tal health prob­lems or a com­bi­na­tion of these, mak­ing it an up­hill task to com­plete the 12-week reg­i­men.

Vin­cent Lo Re Perel­man

This cat­e­go­ry of pa­tient, there­fore, could ben­e­fit from the short­er 8-week treat­ment pe­ri­od, he said.

“The re­al­i­ty is that we’ve treat­ed many pa­tients who have been wait­ing for he­pati­tis C ther­a­py,” he said. “The pa­tients that are now of­ten pre­sent­ing for eval­u­a­tion and con­sid­er­a­tion for treat­ment of­ten have many oth­er co-mor­bidi­ties, so­cial chal­lenges that make even once-dai­ly ther­a­py dif­fi­cult to ad­here to.”

Mavyret de­buted in Au­gust 2017 as the cheap­er al­ter­na­tive to Gilead’s $GILD block­buster-yet-wal­let-bust­ing So­val­di, which came in at $84,000 for a three-month reg­i­men. Still, Ab­b­Vie’s op­tion costs $13,200 per 4-weeks or $26,400 for 8-weeks and so on (at the time some forms of the virus were cured with­in that time­frame, oth­ers af­ter 12 or 16 weeks).

Many in­sur­ers were will­ing to pay the price of these treat­ments, as it saved mon­ey in the long run, but some plans such as Med­ic­aid were un­able to pro­vide it to their pa­tients due to fi­nan­cial con­straints. The dis­ease has now be­come a light­ning rod in the ever-heat­ing de­bate around in­no­va­tion and ac­cess — both an ex­am­ple of the kind of cu­ra­tive ther­a­pies the cur­rent R&D sys­tem can pro­duce and the high costs that tend to de­ny ac­cess to those most in need.

Janet Wood­cock FDA

“I would nev­er have thought, ever, in the ’90s, that if some­body came up with a cure for he­pati­tis C, that peo­ple wouldn’t be able to be treat­ed. That was in­con­ceiv­able,” CDER chief Janet Wood­cock told the Break­throughs in Med­i­cine Con­fer­ence this month. “But fast-for­ward to now, and that’s the re­al­i­ty we’re see­ing.”

But oth­ers have point­ed to the hep C cure as an ex­am­ple of the Amer­i­can sys­tem at work, with Gilead ac­tu­al­ly of­fer­ing the drug for less than they and in­de­pen­dent re­searchers said would max­i­mize their prof­it.

Fol­low­ing mod­els first in­tro­duced abroad, drug com­pa­nies have re­cent­ly turned to­ward in­no­v­a­tive meth­ods of mak­ing their prod­ucts avail­able while main­tain­ing prof­it mar­gins. Gilead and Ab­b­Vie have each signed “Net­flix” style agree­ments with Louisiana and Wash­ing­ton, re­spec­tive­ly, to de­liv­er es­sen­tial­ly un­lim­it­ed quan­ti­ties of the drug to prison and Med­ic­aid pa­tients for a flat rate from the state. These mod­els may not lead to low­er prices for the state gov­ern­ment but do pro­vide cost cer­tain­ty. Oth­er states may fol­low suit.

Ex­act sta­tis­tics are hard to come by and the field is chang­ing with each drug and pric­ing de­vel­op­ment, but a 2018 Open Fo­rum In­fec­tious Dis­ease study found 35% of 9,025 tracked pa­tients were pre­scribed an HCV cure but then de­nied cov­er­age. Those with pri­vate in­sur­ance were the most like­ly to be de­nied.

“Un­for­tu­nate­ly for many pa­tients,” wrote an­oth­er group of re­searchers this year in the Amer­i­can Jour­nal of Med­i­cine, cit­ing the OFID study among oth­ers, “their state of res­i­dence is the de­ci­sive fac­tor for whether they will re­ceive life­sav­ing treat­ment.”

So­cial im­age: Ab­b­Vie

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

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This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

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For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
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FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

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Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.