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

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.