As gener­ic en­try looms for its cash cow, Jazz wins FDA nod for fol­low-up sleep dis­or­der drug

Af­ter a 3-month de­lay at­trib­uted to la­belling dis­cus­sions, Jazz Phar­ma­ceu­ti­cals $JAZZ has notched an ap­proval for its wake-pro­mot­ing drug sol­ri­amfe­tol, now chris­tened Sunosi.

Like Xyrem — the block­buster nar­colep­sy drug that ac­counts for the bulk of Jazz’s sales — Sunosi is de­signed to treat ex­ces­sive day­time sleepi­ness for pa­tients with ob­struc­tive sleep ap­nea (OSA) as well as nar­colep­sy. In 2018, Xyrem sales reg­is­tered at $1.4 bil­lion, trans­lat­ing to over 70% of to­tal rev­enue. But with Sunosi in tow, to­tal prod­uct sales are ex­pect­ed to jump sub­stan­tial­ly.

Jazz bought Sunosi from Aer­i­al Phar­ma in 2014 af­ter the drug had passed muster in 2 Phase II tri­als for $125 mil­lion up­front. It’s the first dual-act­ing dopamine and nor­ep­i­neph­rine re­up­take in­hibitor to be ap­proved by the FDA, and al­though the com­pa­ny is un­clear as to how it works, the drug has im­proved pa­tients’ over­all clin­i­cal con­di­tion com­pared to a place­bo across four Phase III stud­ies.

Bruce Cozadd

“With this ap­proval, a new, day­time med­i­cine that can pro­vide sus­tained wake­ful­ness through­out the day will be avail­able for pa­tients,” CEO Bruce Cozadd said in a state­ment. “The FDA ap­proval of Sunosi al­so rep­re­sents an im­por­tant mile­stone for Jazz as we con­tin­ue to of­fer new treat­ment op­tions that ad­dress un­met needs for peo­ple liv­ing with chron­ic, and of­ten de­bil­i­tat­ing, sleep dis­or­ders.”

Sunosi is ap­proved with once-dai­ly dos­es of 75 mg and 150 mg for pa­tients with nar­colep­sy and dos­es of 37.5 mg, 75 mg, and 150 mg for pa­tients with OSA — but no­tably not 300 mg, Umer Raf­fat of Ever­core ISI wrote in a note.

[In] our opin­ion, not get­ting 300 mg ap­proved might be the sin­gle most im­por­tant thing that makes this la­bel as clean as it is. As you scroll to page 8 of (the) la­bel, there is a ta­ble on BP and HR da­ta on dos­es in­clud­ing 300 mg dose — and you can clear­ly see why FDA did NOT ap­prove that dose de­spite bet­ter ef­fi­ca­cy. In fact, la­bel men­tions “dosages above 150 mg do not con­fer in­creased ef­fec­tive­ness suf­fi­cient to out­weigh AE”.

By not hav­ing 300 mg, the BP and HR events mere­ly be­came a warn­ing, and NOT a black­box. And…al­though la­bel rec­om­mends as­sess­ing BP and HR be­fore ini­ti­at­ing and dur­ing treat­ment, (the) re­al­i­ty is that many nar­colep­sy and OSA pa­tients have mul­ti­ple CV risk fac­tors

Jazz is wait­ing for a DEA sched­ul­ing be­fore it can launch the drug, a process that’s es­ti­mat­ed to take 90 days. Mean­while, the com­pa­ny will be hir­ing 50 more sales reps for their 95-mem­ber sleep team, ac­cord­ing to Cowen an­a­lysts.

By mak­ing some cross-tri­al com­par­isons be­tween Sunosi and cur­rent stan­dard of care Provig­il/Nu­vig­il (which re­cent­ly went gener­ic), they gave the Jazz drug a ring­ing en­dorse­ment as a best-in-class agent.

“Im­por­tant­ly, our con­sul­tant checks and sur­vey re­sults in­di­cate that Sunosi ap­pears to be a need­ed treat­ment op­tion and a like­ly $500 mil­lion+ long du­ra­tion prod­uct,” they wrote. “The bot­tom line is that sol­ri­amfe­tol ap­pears to be the most ef­fec­tive wake-pro­mot­ing agent ever de­vel­oped for EDS.”

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.


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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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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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.

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.

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.