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.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.

Break­through sta­tus and promise of a speedy re­view ar­rives for Op­di­vo/Yer­voy com­bi­na­tion as Bris­tol-My­ers bites at Bay­er

Its frontline and single-agent aspirations have been set back, but Bristol-Myers Squibb just took a big step forward in its efforts to apply its checkpoint inhibitor Opdivo to liver cancer. The FDA has granted breakthrough status and priority review to a combination, second-line treatment.

The designation is for Opdivo (nivolumab) in combination with Yervoy (ipilimumab),  for treating advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. The PD-L1 drug was already approved as a single-agent, second-line treatment for HCC. A PDUFA date was set for March 10, 2020 — just 4 months from now.

Third time un­lucky: Lipocine's lat­est quest to mar­ket their oral testos­terone drug snubbed again by FDA

Lipocine’s latest attempt at securing approval for its oral testosterone drug has fizzled yet again.

The Utah-based drug developer on Monday said the FDA has spurned its marketing application, indicating that some efficacy data on the drug, Tlando, was not up to scratch to treat male hypogonadism, a condition characterized by low production of the hormone testosterone, which is responsible for maintaining muscle bulk, bone growth, and sexual function.

UP­DAT­ED: De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

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Roche steers Gazy­va in­to a new PhI­II pro­gram af­ter com­bo shows promise in lu­pus nephri­tis study

Roche is working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with severe kidney disease associated with lupus after a combination approach helped patients in a mid-stage study.

The 125-patient NOBILITY trial evaluated Gazyva, combined with standard-of-care treatment mycophenolate mofetil or mycophenolic acid and corticosteroids, versus standard treatment alone. The combo met the main goal of inducing a statistically superior complete renal response (CRR) of 40% at week 76, versus 18% in patients given standard treatment, Roche said.