Got­tlieb: 'End the shenani­gan­s' on slow-play­ing REMS to de­lay gener­ic drug com­pe­ti­tion

FDA com­mis­sion­er Scott Got­tlieb yes­ter­day called on brand name drug com­pa­nies to “end the shenani­gans” that re­strict gener­ic drug com­pe­ti­tion, specif­i­cal­ly point­ing to the tac­tic of slow-play­ing shared Risk Eval­u­a­tion and Mit­i­ga­tion Strate­gies (REMS) terms in­tend­ed to pre­vent gener­ic man­u­fac­tur­ers from pur­chas­ing drug prod­ucts need­ed to run bioe­quiv­a­lence or bioavail­abil­i­ty stud­ies that FDA ap­proval re­quires.

The re­marks came at an Fed­er­al Trade Com­mis­sion pan­el on pre­scrip­tion drug com­pe­ti­tion and co­in­cid­ed with new FDA draft guid­ance de­signed to curb the de­lay tac­tic, mak­ing it eas­i­er to im­ple­ment a share a sin­gle shared REMS.

“Cur­rent­ly, un­der a shared sys­tem REMS, mul­ti­ple ap­pli­cants need to co­or­di­nate the sub­mis­sion of iden­ti­cal REMS-re­lat­ed doc­u­ments for their re­spec­tive ap­pli­ca­tions. This draft guid­ance ex­plains the FDA’s cur­rent think­ing that the use of a sin­gle Drug Mas­ter File (DMF) for shared sys­tem REMS sub­mis­sions will al­low man­u­fac­tur­ers with prod­ucts in shared sys­tem REMS pro­grams to sub­mit one col­lec­tive set of files to the agency. This is a first step to­ward ad­di­tion­al ac­tions FDA plans to take to mak­ing shar­ing a sin­gle REMS eas­i­er,” Got­tlieb said.

He not­ed that he hopes this new REMS doc­u­ment tem­plate will stream­line the draft­ing and re­view of shared REMS.

Pan­el Dis­cus­sion

Sev­er­al speak­ers fol­low­ing Got­tlieb echoed his con­cerns on com­pa­nies gam­ing the sys­tem to main­tain mo­nop­o­lies that keep drug prices high.

Rut­gers law pro­fes­sor Michael Car­ri­er dis­cussed pay-for-de­lay agree­ments — where com­pa­nies are paid to keep their com­peti­tor gener­ics from the mar­ket; prod­uct hop­ping where prod­ucts are re­for­mu­lat­ed and pa­tients are mi­grat­ed to a new prod­uct be­fore com­peti­tors are mar­ket­ed; cit­i­zen pe­ti­tions — which are meant to raise le­git­i­mate con­cerns about fol­low-on prod­ucts, but of­ten come near the end of the prod­uct’s ex­clu­siv­i­ty (though 98% are de­nied by FDA); and the REMS re­stric­tions Got­tlieb men­tioned.

As­so­ci­ate pro­fes­sor of med­i­cine at Har­vard Med­ical School Aaron Kessel­heim al­so dis­cussed oth­er fac­tors im­pact­ing gener­ic drug up­take, in­clud­ing ad­ver­tis­ing and pro­mo­tions for brand­ed prod­ucts, pa­tient and physi­cian skep­ti­cism of the qual­i­ty of gener­ic drugs, cost and avail­abil­i­ty is­sues, and oth­er hur­dles to pre­vent com­pe­ti­tion, such as patents cov­er­ing sec­ondary parts of phar­ma­ceu­ti­cals.

Stephen Schon­delmey­er, pro­fes­sor and head of the de­part­ment of phar­ma­ceu­ti­cal care and health sys­tems at the Uni­ver­si­ty of Min­neso­ta, men­tioned that ac­tive phar­ma­ceu­ti­cal in­gre­di­ent (API) con­tracts can lim­it or ma­nip­u­late com­pe­ti­tion and need to be ex­am­ined fur­ther. He of­fered the ex­am­ple of the atenolol short­age as a case where it’s un­clear what the cause of the short­age is.

Chip Davis, pres­i­dent of the gener­ic drug lob­by­ing group AAM, al­so ex­plained how gener­ic drug com­pa­nies are con­sol­i­dat­ing as there are es­sen­tial­ly three large pur­chas­ing con­sor­tia con­trol­ling 90% of the mar­ket.

“Let me be clear: Our mem­bers are not the on­ly ones rais­ing sus­tain­abil­i­ty con­cerns. Pur­chasers are re­in­forc­ing these de­fla­tion­ary trends too,” Davis said. “Sim­ple eco­nom­ics dic­tates that you won’t have 10 to 20 gener­ic com­pa­nies sup­port­ing three buy­ers.”

In terms of so­lu­tions, Kessel­heim was the on­ly pan­elist to call on FDA to con­sid­er a pol­i­cy where­by gener­ic drugs could be im­port­ed more sys­tem­at­i­cal­ly to en­large the US mar­ket. He al­so raised the pos­si­bil­i­ty that for cer­tain niche mar­kets, the US gov­ern­ment could of­fer pur­chas­ing con­tracts to al­low for guar­an­teed de­mand, which is al­ready done with chil­dren’s vac­cines.

Bern­stein se­nior an­a­lyst Ron­ny Gal al­so urged the FTC to put out its think­ing on phar­ma­ceu­ti­cal com­pa­ny-ne­go­ti­at­ed con­tracts with phar­ma­cy ben­e­fit man­agers, as sev­er­al such cas­es will be de­cid­ed over the next 24 months.

Oth­er pan­els on Wednes­day dis­cussed the role of PBMs and group pur­chas­ing or­ga­ni­za­tions in the sup­ply chain, as well as steps to en­cour­age en­try and ex­pand ac­cess through low­er prices.

Im­age: Scott Got­tlieb AP Im­ages

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took at 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Image: Chris Varma. Frontier

UP­DAT­ED: Chris Var­ma un­veils MP­M's lat­est start­up — eye­ing 'un­drug­gable' can­cer tar­gets and pow­ered by ma­chine learn­ing, $67M

Two years af­ter MPM Cap­i­tal en­list­ed Chris Var­ma on its busy on­col­o­gy team, the for­mer en­tre­pre­neur-in-res­i­dence is un­veil­ing his first ven­ture project out of his new stomp­ing grounds in the Bay Area: Fron­tier Med­i­cines.

For Var­ma, who’s al­so co-found­ed Blue­print Med­i­cines and built com­pa­nies at Third Rock and Flag­ship, this marks an­oth­er op­por­tu­ni­ty to ap­ply some cut­ting-edge sci­ence to “sev­er­al of the most im­por­tant and dif­fi­cult tar­gets in can­cer” — tar­gets that oth­ers have tried to tack­le with more clas­si­cal meth­ods and failed. The launch round comes in at $67 mil­lion, which should go some way in scaf­fold­ing a pre­clin­i­cal pipeline and push one or more as­sets in­to the clin­ic three years from now, he tells me.

The top 15 mega-deals in bio­phar­ma: Ab­b­Vie and Bris­tol-My­ers ac­qui­si­tions stir fresh de­bate over what's too big to buy

The debate over what’s too big to buy in biotech is back. A number of top analysts went right after AbbVie’s rationale for the Allergan deal today, just as Bristol-Myers Squibb stirred immediate debate over the worth and wisdom of acquiring Celgene.

To help provide some added context to this discussion, we asked DealForma chief Chris Dokomajilar to look over the past decade of major M&A in biopharma to decipher the top 15 plays.

The new numbers, unadjusted for inflation, harken back to the days of the Pfizer-Wyeth buyout and Merck’s decision to absorb Schering-Plough — both triggered in 2009. The heat over those acquisitions made the big pharma mega-deal highly unpopular for most everyone — except Pfizer — as industry leaders swore off almost all but the handy bolt-on acquisition.

Until recently.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

SQZ, Ery­tech kick off $57M cell ther­a­py part­ner­ship; Jean-Paul Kress lands new CEO gig at Mor­phoSys

→ In a mar­riage of two tech­nolo­gies meant to make cell ther­a­pies more pow­er­ful, SQZ Biotech is team­ing up with France’s Ery­tech Phar­ma for a col­lab­o­ra­tion, with $57 mil­lion re­served for the first project and $50 mil­lion for each sub­se­quent ap­proval (prod­uct or in­di­ca­tion). Hav­ing ac­cess to Ery­tech’s method of fash­ion­ing ther­a­peu­tics from red blood cells, the Cam­bridge, MA-based com­pa­ny said, will am­pli­fy SQZ’s cell en­gi­neer­ing ca­pa­bil­i­ties and al­low them to de­vleop a new class of im­munomod­u­la­to­ry ther­a­pies. Its own tech — so far ap­plied in can­cer but al­so has po­ten­tial in di­a­betes — tem­po­rary dis­rupts the cell mem­brane by squeez­ing the cell, thus cre­at­ing a brief win­dow for tar­get ma­te­ri­als such as anti­gens to en­ter.