Akcea, Io­n­is win over most FDA ex­perts to their rare dis­ease drug volane­sors­en, vast­ly im­prov­ing odds of suc­cess

Akcea Ther­a­peu­tics $AK­CA faced a tough up­hill bat­tle in search of an ap­proval for their rare dis­ease drug volane­sors­en to­day — and they won.

The FDA ad­vi­so­ry com­mit­tee vote on the drug — spun out from Io­n­is $IONS — was 12 for, 8 against. The FDA, of course, doesn’t have to go along. But for a rare dis­ease, they usu­al­ly do — par­tic­u­lar­ly un­der this ad­min­is­tra­tion.

Akcea’s shares shot up 20% in pre-mar­ket trad­ing on Fri­day, start­ing the day with a $1.8 bil­lion mar­ket cap, while Io­n­is saw a spike of 5%.

FDA reg­u­la­tors didn’t make the task very easy, high­light­ing their deep con­cerns with the sud­den plunge in platelets that af­flict­ed a num­ber of pa­tients through the clin­i­cal tri­als for the drug. In­ves­ti­ga­tors nev­er found a re­li­able way to screen for throm­bo­cy­tope­nia in the tri­al work, and their in­ter­nal re­view plant­ed a red flag on the fact that doc­tors pre­scrib­ing this drug would al­so like­ly be blind­sided by un­ex­pect­ed cas­es.

Io­n­is re­port­ed a lit­tle more that a year ago that it had achieved its ef­fi­ca­cy end­point in Phase III. But five pa­tients were forced out of the tri­al due to a threat­en­ing de­cline in platelet counts. Grade 4 throm­bo­cy­tope­nia oc­curred in three pa­tients, which end­ed af­ter they stopped dos­ing. There were no with­drawals due to platelet counts af­ter the com­pa­ny be­gan mon­i­tor­ing the side ef­fect.

If it does get an OK, those facts on safe­ty are like­ly to haunt the com­pa­ny’s sales ef­forts.

In its fa­vor, though, was a sol­id set of da­ta demon­strat­ing the drug’s ef­fect in reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome — but with­out the clear clin­i­cal im­pact that would have helped push this drug over the top with big­ger num­bers in its fa­vor.

“We thank all the mem­bers of the Com­mit­tee for their time in con­duct­ing a thor­ough and thought­ful re­view of Waylivra, a new po­ten­tial first and on­ly ther­a­py for peo­ple with fa­mil­ial chy­lomi­crone­mia syn­drome, or FCS. FCS is ul­tra-rare, se­vere and po­ten­tial­ly fa­tal with no ther­a­peu­tic op­tions. The da­ta, in­clud­ing re­sults from two phase three clin­i­cal tri­als, demon­strate clear im­prove­ment on sev­er­al im­por­tant mea­sures of dis­ease in these pa­tients. We be­lieve that these re­sults pro­vide strong sup­port to make Waylivra avail­able to treat peo­ple with FCS,” said Paula Soteropou­los, chief ex­ec­u­tive of­fi­cer of Akcea Ther­a­peu­tics. “We look for­ward to work­ing with the FDA to com­plete the fi­nal stages of reg­u­la­to­ry re­view for Waylivra. We are com­mit­ted to the FCS com­mu­ni­ty and will con­tin­ue to fo­cus on bring­ing Waylivra to peo­ple suf­fer­ing with this dev­as­tat­ing dis­ease.”

 

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.