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.


Zachary Brennan

managing editor, RAPS

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.