The next Em­flaza: Ac­er Ther­a­peu­tics looks to take a page out of Marathon's book

Last year, Marathon Phar­ma­ceu­ti­cals took an in­ex­pen­sive steroid avail­able for sale in the EU and Cana­da and brought it through the US FDA ap­proval process be­fore trum­pet­ing an $89,000 an­nu­al price.

The move, crit­i­cized by law­mak­ers and oth­ers, of­fered a test case for how a com­pa­ny can use FDA’s ap­proval sys­tem and the US pric­ing sys­tem to reap out­sized re­wards with­out do­ing any of the de­vel­op­ment work.

Frank David

Now, ac­cord­ing to a new preprint from phar­ma­ceu­ti­cal con­sul­tants at the firm Phar­mag­el­lan, an­oth­er com­pa­ny called Ac­er Ther­a­peu­tics, chaired by the cur­rent CEO of Mar­tin Shkre­li’s old com­pa­ny Retrophin, ac­quired celipro­lol, a cheap be­ta block­er long avail­able out­side the US but nev­er sub­mit­ted for FDA ap­proval. Ac­er has said it will seek FDA ap­proval for the drug this year.

“If Ac­er launch­es Ed­si­vo [celipro­lol] at a high US price (a log­i­cal bet), this will be a reprise of last year’s Em­flaza to-do,” Phar­mag­el­lan man­ag­ing di­rec­tor Frank David said. “Ac­er bought the da­ta rights and ob­tained or­phan des­ig­na­tion, but has not con­duct­ed fur­ther tri­als as far as we can tell.”

An Ac­er ex­ec­u­tive told Fo­cus via email: “No com­ment.”

What Can be Done?

Look­ing to ad­vance the dis­cus­sion on how to pre-emp­tive­ly iden­ti­fy and block such be­hav­ior, David and Richa Dix­it said “al­ter­ations to FDA’s ex­ist­ing rules on per­son­al im­por­ta­tion” may be one so­lu­tion.

“Re­fin­ing these cri­te­ria to al­low Amer­i­cans to im­port gener­ics in cer­tain sit­u­a­tions where a drug with the same ac­tive in­gre­di­ent is al­ready avail­able in the US – for ex­am­ple, if the gener­ic was launched abroad be­fore the first FDA ap­proval – would per­mit con­tin­ued im­por­ta­tion of drugs like de­flaza­cort and celipro­lol, and thus elim­i­nate the in­cen­tive for fu­ture com­pa­nies to take a sim­i­lar ap­proach,” they write.

And though FDA has been re­sis­tant in the past to al­ter­ing im­por­ta­tion or re-im­por­ta­tion poli­cies out of sup­ply chain safe­ty con­cerns, David told Fo­cus he thinks these are unique sit­u­a­tions.

“We al­ready al­low pa­tients to im­port drugs un­avail­able in the US for per­son­al use (al­though I reck­on this is cum­ber­some, and it’s ob­vi­ous­ly not cov­ered by pay­ers),” David said via email. “So in prin­ci­ple, FDA al­ready ac­knowl­edges it’s ap­pro­pri­ate to have a path­way for pa­tients to ac­cess drugs from abroad that aren’t ap­proved here, and ac­cepts the trade­offs. The is­sue with celipro­lol and de­flaza­cort is that un­der our cur­rent poli­cies, the abil­i­ty to per­son­al­ly im­port halts when/if the drug is ap­proved by FDA. That’s cer­tain­ly ap­pro­pri­ate in the case of on-patent meds, but here we’re talk­ing about drugs that have al­ready gone gener­ic.”

In ad­di­tion, drug pric­ing scan­dals tend to get lumped to­geth­er. But the Em­flaza ex­am­ple should not be mis­con­strued as the same as say, Shkre­li’s old com­pa­ny Tur­ing Phar­ma­ceu­ti­cals ac­quir­ing Dara­prim (pyrimethamine) and jack­ing up the price over night.

“Here, we fo­cused on a nar­row sub­set, epit­o­mized by Em­flaza: old, cheap drugs on­ly avail­able in the EU that are then brought to the US by third par­ties and get the pric­ing and patent ben­e­fits of ‘in­no­va­tor’ drugs. These aren’t go­ing to be com­mon – we on­ly found one loom­ing ex­am­ple in our work! – but they’re unique com­pared with dara­prim, etc., and so­lu­tions to those lat­ter sit­u­a­tions aren’t go­ing to pre­vent fu­ture Em­flaza-like sce­nar­ios,” David said.


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 news@raps.org for more in­for­ma­tion.

Author

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.

Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

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.

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.

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.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

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 a 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.

Endpoints News

Basic subscription required

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

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.