Got­tlieb seeks new com­par­a­tive ap­proval stan­dard for opi­oids

With just a week left be­fore he de­parts from the FDA, Com­mis­sion­er Scott Got­tlieb on Thurs­day told the Sen­ate Ap­pro­pri­a­tions Com­mit­tee that his agency is in­ter­est­ed in Con­gress cre­at­ing a new stan­dard for opi­oid ap­provals where­by those seek­ing ap­proval would be com­pared to opi­oids al­ready on the mar­ket.

“For this to work, the FDA be­lieves that there should be a pre­mar­ket demon­stra­tion that a new opi­oid is su­pe­ri­or to an al­ready-ap­proved opi­oid or opi­oid-con­tain­ing drug for the same gen­er­al in­di­ca­tion. The stan­dard should be that a new opi­oid or opi­oid-con­tain­ing drug pro­vides a sig­nif­i­cant ad­van­tage rel­a­tive to an al­ready-ap­proved opi­oid or opi­oid-con­tain­ing drug for the same gen­er­al in­di­ca­tion in terms of greater ef­fi­ca­cy or greater safe­ty,” he said.

Drugs that meet this new stan­dard could re­ceive break­through or some oth­er new des­ig­na­tion to fur­ther pri­or­i­tize their re­view. “Even­tu­al­ly, Con­gress could con­sid­er how oth­er in­cen­tives, in­clud­ing ex­clu­siv­i­ty pe­ri­ods, might help dri­ve de­vel­op­ment to­ward drugs with safer at­trib­ut­es,” Got­tlieb added.

While ac­knowl­edg­ing that opi­oids need to be treat­ed dif­fer­ent­ly from oth­er prod­ucts, he al­so said FDA is work­ing to im­ple­ment dif­fer­ent pro­vi­sions from the Sup­port Act, in­clud­ing new re­quire­ments for longer ef­fec­tive­ness stud­ies and oth­er au­thor­i­ties that al­low FDA to re­quire cer­tain pack­ag­ing to be made avail­able for opi­oids that pose a se­ri­ous risk of abuse or over­dose.

“The ques­tion is long-term safe­ty when these drugs are used chron­i­cal­ly, in par­tic­u­lar whether ef­fi­ca­cy de­clines and if that cre­ates safe­ty is­sues,” Got­tlieb said.

But on the ques­tion of whether any com­pa­ny is close to bring­ing a non-opi­oid painkiller to mar­ket, Got­tlieb said they’re in the ear­ly stages of de­vel­op­ment and it’s dif­fi­cult to pre­dict how quick­ly such a prod­uct would come to mar­ket.

He al­so did not seem to think the in­flux of il­lic­it opi­oids was un­der con­trol. “I don’t know that I can say we have a han­dle on it or an un­der­stand­ing of the full scope,” he added.

In ad­di­tion to the opi­oids dis­cus­sion, Sen. Patrick Leahy (D-VT) asked Got­tlieb about the agency’s frame­work for ap­prov­ing biosim­i­lar in­sulins, which is shift­ing in 2020 as prod­ucts ap­proved un­der new drug ap­pli­ca­tions are tran­si­tioned to bi­o­log­ics li­cens­es.

Got­tlieb said he thought that un­der the new biosim­i­lars path­way, “we’ll see much more com­pe­ti­tion” for in­sulins, as it will be eas­i­er to get to mar­ket and com­pete as in­ter­change­able prod­ucts. He ac­knowl­edged there may be one or two ap­pli­ca­tions that get caught and have to be re­sub­mit­ted be­cause of the shift, but he in­sist­ed there will be “vig­or­ous ac­tiv­i­ty un­der the new frame­work.”

He al­so said he hopes Con­gress would reau­tho­rize the im­por­tant biosim­i­lar ap­proval path­way, which is part of the Af­ford­able Care Act, if the ACA were in­val­i­dat­ed.

In his writ­ten tes­ti­mo­ny, Got­tlieb al­so dis­cussed trans­form­ing med­ical de­vice safe­ty and cy­ber­se­cu­ri­ty, med­ical coun­ter­mea­sures and drug com­pound­ing.


Im­age cred­it: Shut­ter­stock

First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Ugur Sahin, BioNTech CEO

Covid-19 roundup: Pfiz­er-backed BioN­Tech plans to seek FDA OK for a new vac­cine 'as ear­ly as' Oc­to­ber — ahead of the elec­tion

BioNTech execs say they’re on track to get their late-stage data on a Covid-19 vaccine — partnered with Pfizer — into the hands of regulators as early as October.

In their Q2 release Tuesday morning, the biotech reported that investigators could have late-stage data as early as October, and they won’t be wasting any time in hustling that over to the FDA.

“I am incredibly proud of our team, who has worked tirelessly to initiate our BNT162 Phase 2b/3 trial in record time and put us in a position to seek regulatory review as early as October of this year, if our trials are successful,” said Ugur Sahin, BioNTech’s CEO and co-founder.

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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