Sanofi's con­tro­ver­sial dengue vac­cine wins FDA ap­proval, with a raft of caveats

Sanofi’s con­tro­ver­sial dengue vac­cine, which took two decades to de­vel­op, has made it across the FDA fin­ish line — but it wasn’t pret­ty. Less than a month af­ter a pan­el of ex­perts de­liv­ered mixed re­views for the shot, on Wednes­day, the US health reg­u­la­tor sanc­tioned the use of Deng­vax­ia as the first-ever prod­uct to pre­vent the mos­qui­to-borne dis­ease in cer­tain in­di­vid­u­als who have pre­vi­ous­ly con­tract­ed dengue and who live in ar­eas rife with the dis­ease.

The FDA has lim­it­ed the use of the vac­cine in in­di­vid­u­als aged 9 to 16 who have lab­o­ra­to­ry-con­firmed pre­vi­ous dengue in­fec­tion and live in ar­eas where the dis­ease is en­dem­ic, such as Puer­to Ri­co, the US Vir­gin Is­lands and Amer­i­can Samoa. Orig­i­nal­ly, Sanofi $SNY had sought to se­cure ap­proval for peo­ple aged 9 through 45, but the FDA ad­vi­so­ry pan­el last month foiled that bid, on­ly agree­ing to rec­om­mend its use in chil­dren.

Deng­vax­ia — which is ad­min­is­tered as three sep­a­rate in­jec­tions — was found to be rough­ly 76% ef­fec­tive in pre­vent­ing symp­to­matic dengue dis­ease in in­di­vid­u­als 9 through 16 years of age who pre­vi­ous­ly had lab­o­ra­to­ry-con­firmed dengue dis­ease, the FDA not­ed. But in the Unit­ed States and its ter­ri­to­ries, no avail­able tests have been cleared by the FDA to de­ter­mine a pre­vi­ous dengue in­fec­tion, Sanofi said.

The French drug­mak­er once had block­buster dreams for the world’s first dengue vac­cine, but that hope was mas­sa­cred af­ter it was forced to con­cede in late 2017 that Deng­vax­ia could en­hance the risk of se­vere dengue in chil­dren who had nev­er been ex­posed to the virus — on­ly af­ter the Philip­pines used $70 mil­lion worth of the shot in a mass vac­ci­na­tion cam­paign for 800,000 chil­dren.

But even be­fore the Philip­pines’ pub­lic health cri­sis, ex­perts had warned that vac­ci­na­tion with Deng­vax­ia in those who had not al­ready been in­fect­ed by one of the four dif­fer­ent serotypes of the virus faced a po­ten­tial­ly life-threat­en­ing fever up­on a sub­se­quent in­fec­tion. This is be­cause their bod­ies would like­ly treat the first re­al dengue in­fec­tion as their sec­ond, thanks to the vac­cine. Ac­cord­ing to the FDA, rough­ly 95% of all se­vere/hos­pi­tal­ized cas­es of dengue are as­so­ci­at­ed with sec­ond dengue virus in­fec­tion.

In March, Philip­pine of­fi­cials said they were prepar­ing crim­i­nal charges against Sanofi, in con­nec­tion with 10 deaths they said were linked to the vac­cine. Sanofi op­posed their find­ings.

The virus is wide­spread across the trop­ics. Se­vere dengue was first rec­og­nized in the 1950s dur­ing dengue epi­demics in the Philip­pines and Thai­land, cur­rent­ly af­fects most Asian and Latin Amer­i­can coun­tries, and is con­sid­ered a lead­ing cause of hos­pi­tal­iza­tion and death among chil­dren and adults in these re­gions, ac­cord­ing to the WHO. The CDC es­ti­mates that each year, about 400 mil­lion dengue virus in­fec­tions oc­cur glob­al­ly. The dengue virus is car­ried by fe­male Aedes ae­gyp­ti mos­qui­toes (and to a less­er ex­tent, Ae. al­bopic­tus)the same species that al­so trans­mits chikun­gun­ya, yel­low fever and Zi­ka.

The French drug­mak­er has earned a trop­i­cal dis­ease pri­or­i­ty re­view vouch­er — grant­ed to drug­mak­ers to re­ward drug/vac­cine de­vel­op­ment for trop­i­cal dis­eases that typ­i­cal­ly don’t get much at­ten­tion — in con­nec­tion with the ap­proval. Some of these vouch­ers have been pre­vi­ous­ly sold for hun­dreds of mil­lions of dol­lars.

Deng­vax­ia has al­ready been cleared for use in 19 coun­tries, and last year was ap­proved by the Eu­ro­pean Union for in­di­vid­u­als aged 9 to 45 years who have been in­fect­ed with dengue virus be­fore and who live in ar­eas where the in­fec­tion is en­dem­ic.

Japan’s Take­da has a ri­val dengue vac­cine in late-stage de­vel­op­ment.

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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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.

Fresh analy­sis spot­lights car­dio ben­e­fit of J&J's In­vokana in di­a­betes pa­tients with­out his­to­ry of CV dis­ease

In­vokana sales may be mut­ed, but the di­a­betes drug is set to get some love af­ter its mak­er J&J un­veiled da­ta at the Amer­i­can Di­a­betes As­so­ci­a­tion meet­ing on Tues­day sug­gest­ing the med­i­cine can con­fer a car­dio­vas­cu­lar ben­e­fit in pa­tients who do not have pre­ex­ist­ing CV dis­ease.

Back in April, J&J had re­port­ed that in the late-stage CRE­DENCE study, the SGLT2 drug scored a 30% re­duc­tion in the risk of a com­pos­ite of ail­ments: a pro­gres­sion to the dou­bling of serum cre­a­ti­nine, end-stage kid­ney dis­ease and re­nal or car­dio­vas­cu­lar death. In terms of sec­ondary end­points, the drug was al­so found be heart-pro­tec­tive: low­er­ing the risk of CV death and hos­pi­tal­iza­tion for heart fail­ure by 31%, as well as ma­jor ad­verse CV events by 20%. In March, the com­pa­ny sub­mit­ted an ap­pli­ca­tion to ex­pand In­vokana’s la­bel to re­flect its im­pact on chron­ic kid­ney dis­ease.

Sil­i­con Val­ley's most an­tic­i­pat­ed slide deck just dropped. What does it mean for bio­phar­ma's dig­i­tal teams?

These aren’t the typ­i­cal slides you’d see at End­points — no mol­e­cules, clin­i­cal pro­grams, or p-val­ues. In­stead, we’ll talk dig­i­tal and in­ter­net trends, fac­tors that elite glob­al brands — re­gard­less of in­dus­try — must first mea­sure and un­der­stand be­fore de­ploy­ing prod­ucts in­to the world. That’s a con­cept that most of our Big Phar­ma au­di­ence is in tune with. Dig­i­tal aware­ness is key to suc­cess in the dis­cov­ery, de­vel­op­ment, and mar­ket­ing of new bio­phar­ma­ceu­ti­cals, and most of the ma­jors now have a chief dig­i­tal of­fi­cer: No­var­tis, Sanofi, and Pfiz­er, just to name a few.