Scott Got­tlieb has some ideas about block­ing any more would-be Mar­tin Shkrelis from price goug­ing

FDA com­mis­sion­er Scott Got­tlieb wants to stop the next Mar­tin Shkre­li be­fore he, or she, gets start­ed on the next drug price goug­ing scan­dal. And shin­ing a bright light on the drugs most vul­ner­a­ble to pric­ing abuse will be one way to get start­ed.

In tes­ti­mo­ny be­fore the House ap­pro­pri­a­tions sub­com­mit­tee Thurs­day, the new com­mis­sion­er high­light­ed sev­er­al meth­ods that could pre­vent bio­phar­ma com­pa­nies from gam­ing the sys­tem, fo­cus­ing specif­i­cal­ly on “sit­u­a­tions where off-patent drugs lack an ap­proved gener­ic com­peti­tor.” Got­tlieb will get the FDA to pub­lish a list of drugs that are off patent “for which FDA has not ap­proved a sin­gle gener­ic ap­pli­cant.”

Scott Got­tlieb

Got­tlieb wants to flag the po­ten­tial for a re­peat of Shkre­li’s de­ci­sion to buy an old gener­ic, Dara­prim, and then rais­ing the price of the drug overnight by more than 5000% at his com­pa­ny, Tur­ing. Dara­prim had no gener­ic com­pe­ti­tion on the mar­ket, giv­ing Shkre­li a clear and le­gal path to jack­ing up the price.

There are var­i­ous ways to im­prove the gener­ic drug ap­proval process to in­crease com­pe­ti­tion and pre­vent play­ers from gam­ing prices, he says. But it’s clear­ly an is­sue he wants to tack­le ear­ly on.

“We do have sit­u­a­tions where spec­u­la­tors, for lack of a bet­ter word, can come in and buy a low vol­ume gener­ic, jack up the price know­ing that it will take po­ten­tial­ly years for gener­ic com­pe­ti­tion to come on to the mar­ket so they have that sort of an ex­clu­siv­i­ty pe­ri­od,” said the com­mis­sion­er.

Got­tlieb out­lined a three-prong plan to speed up gener­ic drug ap­provals and com­plete­ly elim­i­nate the back­log of ap­pli­ca­tions for knock­offs, hur­ry­ing along cheap­er al­ter­na­tives to brand ther­a­pies that could go a long way to re­duc­ing the coun­try’s spend­ing on phar­ma­ceu­ti­cals.

He told law­mak­ers that he plans to:

— “Cur­tail gam­ing by in­dus­try of our reg­u­la­tions which can ex­tend mo­nop­oly pe­ri­ods be­yond the time frame Con­gress in­tend­ed, hin­der­ing com­pe­ti­tion.”

— “Im­prove the process­es that en­able gener­ic ver­sions of com­plex drugs to be ap­proved for mar­ket­ing.”

— “In­crease the over­all ef­fi­cien­cy of the gener­ic drug re­view process while com­plete­ly elim­i­nat­ing the back­log of gener­ic ap­pli­ca­tions.”

The REMS pro­gram (risk eval­u­a­tion and mit­i­ga­tion strate­gies), he not­ed, has been used to slow gener­ic com­pe­ti­tion, and stream­lin­ing the process by waiv­ing the re­quire­ment that a gener­ic and brand man­u­fac­tur­er share a sin­gle sys­tem for as­sur­ing safe use could be used to elim­i­nate one tool used to slow gener­ics.

In the past, gener­ic man­u­fac­tur­ers have had to sue brand op­er­a­tions which re­fused to ne­go­ti­ate a shared safe­ty sys­tem as a way to block cheap­er com­pe­ti­tion.

Rep. Robert Ader­holt

Got­tlieb al­so sin­gled out the on­col­o­gy di­vi­sion at the FDA for em­brac­ing the break­through ther­a­py pro­gram, speed­ing along new ap­provals. Un­for­tu­nate­ly, he added, not every part of the agency does as well as the on­col­o­gy group, and he in­tends to bring the lag­gards up to speed. In ad­di­tion, he added, the FDA could do more by tak­ing a more pro­gres­sive view of some new tech­nolo­gies, like bio­mark­ers, in as­sess­ing drugs, look­ing at tri­al de­signs to find more ef­fi­cient ways to de­vel­op drugs and adding new tools for ther­a­peu­tic re­views.

It was im­me­di­ate­ly clear that the law­mak­ers of all stripes didn’t care for the Trump ad­min­is­tra­tion’s plans for the 2018 FDA bud­get.

De­moc­rats and Re­pub­li­cans alike wast­ed lit­tle time in crit­i­ciz­ing the Trump ad­min­is­tra­tion’s plan to dou­ble up on in­dus­try user fees while slash­ing the fed­er­al gov­ern­ment’s con­tri­bu­tion to the FDA’s bud­get.

“The agency’s chances of off­set­ting bud­get au­thor­i­ty with user fees face a very stiff up­hill bat­tle in the fu­ture,” not­ed Al­aba­ma Re­pub­li­can Robert Ader­holt. He al­so cit­ed the FDA’s rapid progress in hus­tling along new ap­provals of Keytru­da, which he should help, “uti­liz­ing the lat­est tech­nol­o­gy, make drugs ac­ces­si­ble be­fore a lengthy drug tri­al.”

One plus for the FDA: Got­tlieb not­ed that the FDA is no longer in the grip of a fed­er­al hir­ing freeze im­posed by Pres­i­dent Trump, ac­cord­ing to RAPS. The freeze, he told law­mak­ers, has been lift­ed.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.