Shat­ter­ing an old record, FDA quick­ly OKs pi­o­neer­ing, site-ag­nos­tic can­cer drug larotrec­tinib from Loxo and Bay­er

The FDA has hus­tled up a quick OK for larotrec­tinib, of­fer­ing their stamp of ap­proval for a site-ag­nos­tic can­cer drug from Loxo On­col­o­gy and their part­ners at Bay­er, a prime ex­am­ple of a new wave of on­col­o­gy drugs hit­ting the field.

The la­bel for their drug, now called Vi­t­rakvi, was the first thing to hit Twit­ter af­ter the mar­ket closed, which you can see here.

Stam­ford, CT-based Loxo $LOXO — run by CEO Josh Bilenker — is one of the trail­blaz­ers in per­son­al­ized can­cer ther­a­pies where pa­tients are grouped not by the site of tu­mor de­vel­op­ment but by ge­net­ics — an ap­proach that calls for broad­er se­quenc­ing to ID the ge­net­ic mu­ta­tions un­der­pin­ning each pa­tients’ can­cer. That re­quires lots of work to iden­ti­fy pa­tients. But they have da­ta from a small piv­otal study to back up their break­through.


Specif­i­cal­ly there were 50 pa­tients — 43 adults and 12 chil­dren and adults with TRK fu­sion can­cer — with 12% com­plete and 64% par­tial re­spon­ders. That’s a high rate of suc­cess and it qual­i­fied for a pri­or­i­ty re­view at an agency that likes to press the met­al on new drug OKs — par­tic­u­lar­ly for can­cer.

Now comes the re­al­ly hard part. To find the few thou­sand US pa­tients that could ben­e­fit will re­quire can­cer pa­tients to get their DNA se­quenced, which is un­com­mon. Then theres the price. Bay­er set the whole­sale price at just un­der $400,000 a year, putting the oral drug in the most ex­pen­sive drug cat­e­go­ry. Then there’s a liq­uid for­mu­la­tion avail­able for cer­tain chil­dren at $132,000 a year.

Bay­er is al­so of­fer­ing a mon­ey-back guar­an­tee to pay­ers with as­sur­ances that pa­tient’s out-of-pock­et ex­po­sure will usu­al­ly be lim­it­ed to small sums.

An­drew Berens at Leerink ex­pects to see the num­bers add up over the years.

We see peak Vi­t­rakvi rev­enues in the US of ~$700mn by 2030, with an ad­di­tion­al ~$375mn op­por­tu­ni­ty avail­able for next-gen­er­a­tion TRK in­hibitor LOXO-195, de­signed specif­i­cal­ly to ad­dress re­sis­tance mu­ta­tions that emerge with larotrec­tinib use.

“Its ap­proval re­flects ad­vances in the use of bio­mark­ers to guide drug de­vel­op­ment and the more tar­get­ed de­liv­ery of med­i­cine,” not­ed FDA com­mis­sion­er Scott Got­tlieb, who likes to step in when the agency breaks new ground. “We now have the abil­i­ty to make sure that the right pa­tients get the right treat­ment at the right time. This type of drug de­vel­op­ment pro­gram, which en­rolled pa­tients with dif­fer­ent tu­mors but a com­mon gene mu­ta­tion, wouldn’t have been pos­si­ble a decade ago be­cause we knew a lot less about such can­cer mu­ta­tions.”

A few months af­ter the da­ta ar­rived Bay­er bought in, hand­ing over a $400 mil­lion up­front, with $450 mil­lion in mile­stones for the de­vel­op­ment and first sale of larotrec­tinib, with an­oth­er $200 mil­lion on the ta­ble for LOXO-195. There’s al­so $500 mil­lion on the books for com­mer­cial goals.

Mer­ck was the first to score in this site-ag­nos­tic field, win­ning an FDA OK last year to use Keytru­da against tu­mors that were mi­crosatel­lite in­sta­bil­i­ty-high or char­ac­ter­ized by mis­match re­pair de­fi­cien­cy.

This is the 53rd new drug ap­proval for the FDA’s CDER, which with sev­er­al new bi­o­log­ic OKs this year has shat­tered the record to­tal of 53 drugs ap­proved in 1996. With 5 weeks to go in the year, the agency has plen­ty of time to set a brand new bar for pro­duc­tiv­i­ty in bio­phar­ma R&D.


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.