Com­pared to avoid­ance, Aim­mune's peanut al­ler­gy treat­ment an im­prove­ment over DB­V's prod­uct — ICER

Peanut al­ler­gy treat­ments from spar­ring drug­mak­ers — Aim­mune and DBV Tech­nolo­gies — are in­cre­men­tal­ly ben­e­fi­cial, but their long-term cost-ef­fec­tive­ness will be de­ter­mined by the price at which they are even­tu­al­ly mar­ket­ed, ICER said in a draft re­port pub­lished on Tues­day, which con­clud­ed us­ing as­sumed prices that the lat­ter’s prod­uct, Vi­askin Peanut, was far less of an im­prove­ment over peanut avoid­ance, com­pared to AR101.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly be­com­ing in­flu­en­tial with pay­ers.

For now, peanut al­ler­gies are man­aged by avoid­ance, but the threat of ac­ci­den­tal ex­po­sure can­not be nul­li­fied. Aim­mune’s AR101 and DBV’s Vi­askin Peanut are set to be the pi­o­neer­ing peanut al­ler­gy treat­ments ap­proved by the FDA, but there is “sig­nif­i­cant un­cer­tain­ty about the long-term risks and ben­e­fits” for both ther­a­pies, as each has been stud­ied in place­bo-con­trolled one-year clin­i­cal tri­als, ICER said, not­ing that da­ta from the ex­ten­sion tri­als are sparse.

An FDA ap­proval de­ci­sion for AR101 is ex­pect­ed in Jan­u­ary 2020, while DBV is ex­pect­ed to sub­mit its mar­ket­ing ap­pli­ca­tion lat­er in 2019. The so far un­tapped mar­ket is ex­pect­ed to grow to $4.5 bil­lion in 2027 glob­al­ly, ac­cord­ing to Glob­al­Da­ta.

Aim­mune $AIMT ef­fec­tive­ly leapfrogged DBV $DB­VT when the lat­ter re­scind­ed an ap­pli­ca­tion to mar­ket Vi­askin Peanut patch last year in re­sponse to FDA con­cerns about the state of man­u­fac­tur­ing and qual­i­ty con­trol da­ta sub­mit­ted.

Since nei­ther ther­a­py has been ap­proved yet, ICER con­duct­ed its analy­ses us­ing price as­sump­tions, based on an­a­lyst mod­els, which project AR101 will cost be­tween $5,000 and $10,000 for the first six months of use, and $300 to $400 per month af­ter and that Vi­askin Peanut will cost more than $6,000 for a year’s sup­ply.

Based on these es­ti­mates, ICER as­sumed a place­hold­er cost for AR101 at $350 per month ($6,595 for months 1-6 in­clud­ing clin­i­cal vis­its for dose es­ca­la­tion; $4,200 per year there­after). For Vi­askin Peanut, the in­sti­tute as­sumed a place­hold­er cost of $6,500 per year. That works out to AR101 cost­ing $84,000 over a life­time, and Vi­askin Peanut cost­ing $56,000 over a life­times, ICER said.

“Rel­a­tive to our AR101 pric­ing as­sump­tions based on our con­ver­sa­tions with Aim­mune, we had con­ser­v­a­tive­ly as­sumed the low­er end of the com­pa­ny’s com­mu­ni­cat­ed pric­ing range of $5,000 for the up-dos­ing phase and $5,000 per year there­after ($415/month) – so $7,500 for the first year. All-in, the first year costs ICER us­es for its analy­sis are high­er by about 16%,” Stifel’s Derek Archi­la wrote in a note.

For Vi­askin Peanut, ICER’s as­sumed an­nu­al cost of ther­a­py is 20% high­er than Stifel’s $5,000 as­sump­tion, he added.

ICER based its cost-ef­fec­tive­ness cal­cu­la­tions on qual­i­ty-ad­just­ed-life-years (QALYs), a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life.

Treat­ment with AR101 re­sult­ed in 0.63 in­cre­men­tal QALYs, while treat­ment with Vi­askin Peanut came up rel­a­tive­ly short, re­sult­ing in 0.22 in­cre­men­tal QALYs — when com­pared to no im­munother­a­py treat­ment over a life­time, ICER’s analy­sis sug­gest­ed.

“These ben­e­fits are due to im­proved sub­jec­tive qual­i­ty of life de­spite the rel­a­tive rar­i­ty with which se­ri­ous events oc­cur. The ul­ti­mate val­ue of these prod­ucts will be de­ter­mined by the prices that are set by the man­u­fac­tur­ers and their long-term ef­fec­tive­ness,” ICER con­clud­ed.

While in­for­ma­tive, the re­port is not con­clu­sive, part­ly be­cause a one-year time­frame may not re­flect the en­tire ben­e­fit of a ther­a­py that pro­vides in­creas­ing ef­fi­ca­cy over time such as AR101 or Vi­askin peanut, and no qual­i­ty of life da­ta (good or bad) was fac­tored in­to this analy­sis, Archi­la said.

ICER did ac­knowl­edge that one of the lim­i­ta­tions of its analy­sis is that it as­sumed the util­i­ty of the two peanut al­ler­gy ther­a­pies on the ba­sis of ex­ist­ing da­ta on food al­ler­gies, but not specif­i­cal­ly the peanut al­ler­gy pa­tient pop­u­la­tion, due to “the pauci­ty of pref­er­ence-weight­ed health-re­lat­ed qual­i­ty of life es­ti­mates in food al­ler­gy pa­tients and their care­givers.”

ICER’s as­sess­ment is “pre­ma­ture” and the in­sti­tute’s frame­work does not take in­to ac­count the bur­den care­givers car­ry, in terms of mak­ing ther­a­peu­tic de­ci­sions or mak­ing out-of-pock­et costs for treat­ment, a DBV spokesper­son wrote in an email to End­points News.

“We dis­agree with ICER on many as­pects of its over­all method­ol­o­gy, as well as the tim­ing of this re­port. Be­cause the ICER mod­el re­lies on health eco­nom­ic mea­sure­ments…for which there are no pub­lished da­ta/ev­i­dence for peanut-al­ler­gic pa­tients [as there are no ap­proved FDA-ap­proved treat­ments], we be­lieve the draft re­port find­ings were dri­ven by in­ac­cu­rate clin­i­cal out­comes and cost in­puts…We be­lieve the lack of FDA-ap­proved ther­a­peu­tic op­tions for peanut al­ler­gy and as­so­ci­at­ed ab­sence of health state util­i­ty and long-term treat­ment da­ta, com­bined with pa­tient het­ero­gene­ity, pre­cludes an ac­cu­rate and re­li­able cost-ef­fec­tive­ness as­sess­ment, in­clud­ing by ICER.”

End­points has al­so con­tact­ed Aim­mune for com­ment.


Im­age: Shut­ter­stock

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.

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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Ted Love. HAVERFORD COLLEGE

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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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.

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).

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.

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.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Mer­ck grows Keytru­da in­di­ca­tions with head and neck can­cer ap­proval

Mer­ck’s check­point star is shin­ing a lit­tle brighter to­day with two new ap­provals in head and neck squa­mous cell car­ci­no­ma.

The phar­ma gi­ant first clinched an ac­cel­er­at­ed ap­proval to use Keytru­da in re­cur­rent or metasta­t­ic cas­es in 2016, tar­get­ing pa­tients whose dis­ease pro­gressed on or af­ter plat­inum-based chemother­a­py.

On Tues­day the FDA con­vert­ed it in­to a full ap­proval right at the end of a pri­or­i­ty re­view pe­ri­od, and ad­di­tion­al­ly stamped its OK on Keytru­da as a front­line treat­ment for head and neck can­cer. That cov­ers both monother­a­py (in pa­tients whose tu­mor ex­press PD-L1) and a com­bi­na­tion with plat­inum and flu­o­rouracil, or FU (whole pop­u­la­tion).